Researchers tissue gene targeting. Medical search. Frequent questions

Humanized immune system mice, or models that express human versions of mouse immune genes, can be used to model the human immune system, enabling the evaluation of therapies relevant to human health in an in vivo environment. (genetargeting.com)
It's only a letter, but it's a massive difference in terms of biology and physiology," he said, and could be why therapies targeting 2C receptors have been ineffective. (medscape.com)
Previous attempts to develop therapies by targeting different genes of the Shh pathway have not been entirely successful in preventing secondary tumors. (texaschildrens.org)
CAR-T immune therapies could be effective against solid tumors if the right targets are identified, a new study led by University of Illinois Urbana-Champaign researchers suggests. (illinois.edu)
The researchers successfully deployed CAR-T in a mouse model of ovarian cancer, a type of aggressive, solid-tumor cancer that has eluded such therapies until now. (illinois.edu)
Cronin says the team hopes to engineer the bacteria to deliver gene therapies or to activate systemically administered drugs specifically at tumor sites. (aacrjournals.org)
Learn more about how researchers are exploring somatic instability and DNA repair to design therapies for HD. (hdbuzz.net)
AlveoGene will work on gene therapies for several rare lung conditions, beginning with alpha-1 antitrypsin deficiency. (imperial.ac.uk)
Equally as intriguing are the advances in computing power, nanotechnology and gene editing enabling these new tools and potential therapies. (issuu.com)
The findings provide new insights into the role and importance of exosomes and their targeted gene transcripts, and may help point the way to new drugs or therapies for not just skin diseases, but other disorders in which stem and progenitor cell populations are affected. (scitechdaily.com)
That's changing now as a growing number of clinical trials are beginning to test gene therapies in humans. (singularityhub.com)
The broad spectrum of tissues gives hope that further developments will yield lasting and effective results, especially if gene editing is used in conjunction with current therapies. (hivmanagement.net)
In this article, we will discuss the past, present, and future of vector-based gene therapies. (atacana.com)
This was followed by the US approving its first vector-based gene therapies: Luxturna in 2017 and Zolgensma in 2019 10-12 . (atacana.com)
Now, gene therapies are being developed and tested for several diseases, including genetic disorders, cancers, and infectious diseases 13-15 . (atacana.com)
To date, there have been 331 phase I, II, and III clinical trials to assess the safety and efficacy of AAV-based gene therapies, one of the most actively investigated types of gene therapy, for a plethora of human genetic diseases 13 . (atacana.com)
There are currently two FDA-approved Lentivirus (LV)-based gene therapies, Zynteglo and Skysona, and one Herpes simplex virus-based therapy, Vyjuvek. (atacana.com)
A summary of all current FDA-approved gene therapies is shown in Table 1. (atacana.com)
Before the development of these gene therapies, treatment options were limited, and the diseases often led to severe disability or early death. (atacana.com)
Another significant challenge with gene therapies is the potential for immune responses. (atacana.com)
In work described in today's issue of the Proceedings of the National Academy of Sciences , Kuo's group identified five visually discernable kinds of tumors strongly associated with particular gene-expression profiles that are tied to targeted therapies. (technologyreview.com)
For example, Wen says, oncologists have suspected the connection between tumor appearance and response to therapies targeting tumor blood vessels demonstrated in Kuo's study. (technologyreview.com)
Modular Design of Synthetic Receptors for Programmed Gene Regulation in Cell Therapies. (lu.se)

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms. (genomeweb.com)
Gene silencing is considered a gene knockdown mechanism since the methods used to silence genes, such as RNAi, CRISPR, or siRNA, generally reduce the expression of a gene by at least 70% but do not eliminate it[citation needed]. (wikipedia.org)
Researchers at the Weizmann Institute of Science have now combined two powerful research tools - CRISPR gene editing and single-cell genomic profiling - in a method that may finally help provide answers to these questions and many more. (weizmann-usa.org)
The gene-editing technique CRISPR is already transforming biology research around the world, and its clinical use in humans is just around the corner. (weizmann-usa.org)
Combing CRISPR with the fine resolution of single-cell RNA sequencing can enable researchers to actively tinker with the genes in cells, and then to understand their functions within numerous cell types in a variety of situations. (weizmann-usa.org)
The challenge for Prof. Amit and his group, including lead authors Drs. Jaitin, Ido Yofe, and Assaf Weiner and research student David Lara-Astiaso, was to adapt the CRISPR gene-editing technology so it could be used in combination with single-cell sequencing. (weizmann-usa.org)
CRISPR gene editing has had a big decade. (singularityhub.com)
The CRISPR-Cas9 system has emerged as a revolutionary tool in the field of genetic engineering and gene editing . (biocellsupply.com)
Originating from a bacterial adaptive immune system, CRISPR-Cas9 has quickly gained popularity due to its remarkable ability to target specific genes and make precise modifications to the genome. (biocellsupply.com)
The CRISPR-Cas9 system has revolutionized the field of genetic engineering and gene editing by providing a more efficient, targeted, and versatile method. (biocellsupply.com)
In conclusion, CRISPR-Cas9 has transformed the field of genetic engineering and gene editing. (biocellsupply.com)
CRISPR-Cas9 allows researchers to create disease models using stem cells, providing valuable insights into the mechanisms and progression of various diseases. (biocellsupply.com)
Another significant application of CRISPR-Cas9 in stem cell research is the study of gene function. (biocellsupply.com)
Researchers developed a deep learning model, TIGER, that accurately predicts on- and off-target activity of RNA-targeting CRISPR tools. (neurosciencenews.com)
Then, there is cell/gene modification, where genetic modification techniques such as CRISPR-Cas9 are employed to precisely modify and repair a damaged, endogenous gene in the body 3 . (atacana.com)
Data from two pivotal trials suggest that a single infusion of the CRISPR-based gene therapy exagamglogene autotemcel (exa-cel) can provide a 'functional cure' for patients with transfusion-dependent beta-thalassemia or severe sickle cell disease. (cdc.gov)
CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat disease. (cdc.gov)

The first way - 'gene trapping' - creates random mutations by inserting a piece of DNA into the gene. (progress.org.uk)
Both should result in conditional mutations, ones that can be turned on or off depending on the type and developmental stage of the tissue. (progress.org.uk)
They also noted that, in the future, it may make more sense to identify tumors based on genetic mutations rather than their organ or tissue of origin. (doctorshealthpress.com)
Mutations in the PEX7 gene cause a small percentage of all cases of Refsum disease. (medlineplus.gov)
More than three dozen mutations in the PEX7 gene have been found to cause rhizomelic chondrodysplasia punctata type 1 (RCDP1). (medlineplus.gov)
The PEX7 gene mutations responsible for RCDP1 prevent peroxisomal biogenesis factor 7 from transporting critical enzymes, particularly alkylglycerone phosphate synthase, into peroxisomes. (medlineplus.gov)
Many human diseases are known to be caused in part by specific mutations in human genes. (genetargeting.com)
Not all mutations will influence gene expression, but this approach improves our ability to pinpoint those that do. (uclahealth.org)
Sen said the findings could have particular relevance if future research determines that mutations in exosome genes are linked to skin disorders or other diseases. (scitechdaily.com)
Once mutations in exosome complex genes are identified in either skin diseases or other diseases like pontocerebellar hypoplasia, it may be possible to design drugs targeting these defects. (scitechdaily.com)
By introducing specific genetic modifications, researchers can recreate the genetic mutations associated with a particular disease in stem cells. (biocellsupply.com)
It enables the correction of disease-causing mutations, the creation of disease models, and the study of gene function in stem cells. (biocellsupply.com)
Researchers have reverse-engineered the outlines of a disrupted prenatal gene network in schizophrenia, by tracing spontaneous mutations to where and when they likely cause damage in the brain. (neurosciencenews.com)
Mutations can lead to loss of integrity of a whole pathway, not just of a single gene. (neurosciencenews.com)
Earlier studies had linked spontaneous mutations to non-familial schizophrenia and traced them broadly to genes involved in brain development, but little was known about convergent effects on pathways. (neurosciencenews.com)
only one gene harboring damaging mutations turned up in more than one of the cases, and several patients had damaging mutations in more than one gene. (neurosciencenews.com)
The networks formed by genes harboring these damaging mutations were found to vary in connectivity, based on the extent to which their proteins are co-expressed and interact. (neurosciencenews.com)
The network formed by genes harboring damaging mutations in schizophrenia had significantly more nodes, or points of connection, than networks modeled from unaffected siblings. (neurosciencenews.com)
By contrast, the network of genes harboring non-damaging mutations in affected siblings had no more nodes than similar networks in unaffected siblings. (neurosciencenews.com)
When the researchers compared such network connectivity across different brain tissues and different periods of development, they discovered a notable difference between affected and unaffected siblings: Genes harboring damaging mutations that are expressed together in the fetal prefrontal cortex of people with schizophrenia formed a network with significantly greater connectivity than networks modeled from genes harboring similar mutations in their unaffected siblings at that time in development. (neurosciencenews.com)
Studies using cell and animal models are needed to better understand the normal function of this gene and how mutations or loss of the protein leads to SYS and PWS. (fpwr.org)

Cell type-specific partially methylated domains and differentially methylated domains found in normal cells persist in malignant cells, researchers from Cedars-Sinai Medical Center in Los Angeles and elsewhere report in Genome Biology . (genomeweb.com)
They analyzed the whole-genome bisulfite sequencing profiles of 45 esophageal tumors, representing both the squamous cell carcinoma and adenocarcinoma subtypes, and matched nonmalignant tissue. (genomeweb.com)
In contrast, when genes are knocked out, they are completely erased from the organism's genome and, thus, have no expression. (wikipedia.org)
of Health is launching a program to better understand the function of every human gene and generate a catalog of the … in Cells (MorPhiC) program, managed by the National Human Genome Research Institute, aims to systematically investigate … Fletcher, Ph.D., NHGRI program director in the Division of Genome Sciences. (genome.gov)
Compared to other potential animal model systems, it is relatively easy to manipulate the mouse genome , for example, adding or removing a gene to better understand its role in the body. (genetargeting.com)
One location where expression information for endogenous mouse genes is collected is the Mouse Genome Informatics database . (jax.org)
Researchers have moved us a step closer to having the world's first yeast strain with an entirely synthetic and versatile genome - an advance seen as a future-defining moment in science. (nanowerk.com)
Gene expression offers a molecular signpost pointing scientists in the right direction by narrowing the field and highlighting specific areas of the genome. (uclahealth.org)
A gene mutation accompanied by a change in expression clues us to a hot spot on the genome and directs us where to look next," he added. (uclahealth.org)
Researchers are now exploring new techniques such as base editing and epigenetic modifications, which allow for further control and precision when modifying the genome. (biocellsupply.com)
Its evolution has brought about a new era of scientific possibilities, offering researchers unparalleled control and precision in manipulating the genome. (biocellsupply.com)
The Rosa26 locus in the mouse genome is a "safe harbor" which allows researchers to express genes of interest. (renmab.com)
In contrast, gene targeting allows scientists to either "knockout" a gene of interest or make an insertion - knock-in - at a specific site in the mouse genome. (renmab.com)
In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. (cdc.gov)
In these studies, cells are either removed and edited in tissue culture and then readministered to the patient (ex vivo), or genome editors are packaged within viral vectors or lipid nanoparticles and given intravenously to home to specific tissues (in vivo). (cdc.gov)
The ABCs of Gene Cloning, Third Edition contains updates including a tutorial chapter on gene-vector construction, methodologies on exome sequencing in finding disease genes, revised topics on gene therapy and whole genome sequencing, new developments for gene targeting and genome editing, as well as the current state of next generation sequencing. (lu.se)

In a study in mice, periodontal bone loss was greater in normal mice with more succinate and mitigated in mice deficient in the gene for succinate receptors. (newswise.com)
Scientists have explored several drugs targeting specific receptors although such efforts did not yield effective results for patients with ovarian cancer. (medicalxpress.com)
The researchers did not intend to study PCTPs in particular-they were interested in investigating how nuclear receptors get activated. (anl.gov)

To directly compare the specificity and efficacy of different Cre lines in mediating adipocyte specific recombination, transgenic Cre lines driven by the adipocyte protein 2 (aP2) and adiponectin (Adipoq) gene promoters, as well as a tamoxifen-inducible Cre driven by the aP2 gene promoter (iaP2), were bred to the Rosa26R (R26R) reporter. (diabetesjournals.org)
Many Cre transgenes have expression in unexpected organs and tissues, even when the promoters are thought to be tissue-specific. (jax.org)
Improving specificity through genetic modifications or utilizing tissue-specific promoters can enhance targeting efficiency while minimizing off-target effects. (aliyuncs.com)

Skogholt is a researcher at the Norwegian University of Science and Technology's (NTNU)K.G. Jebsen Center for Genetic Epidemiology and a key figure in the international collaboration. (eurekalert.org)
Although we do not look alike, on a genetic level mouse genes are very similar to our own, making mouse genetic research useful for studying human disease. (genetargeting.com)
Researchers looked through electronic medical records from the UK Biobank, a large-scale database that contains genetic and health information on half a million participants. (medscape.com)
These ES cells allow for the generation of gene‐targeted mice on a C57BL/6 genetic background without the need for multiple backcrossing of mice. (umassmed.edu)
In a study publishing May 21 in the journal Cell , researchers use a genetic database of more than 47,000 people in Estonia to identify a gene linked to thinness that may play a role in resisting weight gain in these metabolically healthy thin people. (bajaprogofficial.com)
The team compared the DNA samples and clinical data of healthy thin individuals with normal-weight individuals and discovered genetic variants unique to thin individuals in the ALK gene. (bajaprogofficial.com)
Standard genetic analysis methods involving single-gene tests are often utilized by inherited disease researchers but these methods alone may be time consuming and expensive to uncover the many variants involved in complex diseases such as Noonan syndrome. (thermofisher.com)
Knowledge about the genetic causes of RASopathies is constantly evolving due to the efforts of clinical researchers like those at IPATIMUP. (thermofisher.com)
The mRNA instructs the cells to produce the Cas9 protein (CRISPR's genetic 'scissors') which then links up with the guide RNA, seeks out the target gene, and snips it. (singularityhub.com)
By harnessing the power of this revolutionary technology, researchers can introduce targeted genetic modifications into stem cells, opening up new possibilities for understanding and treating diseases. (biocellsupply.com)
By precisely editing the DNA of stem cells, researchers can fix genetic abnormalities that contribute to various diseases. (biocellsupply.com)
The system works by targeting the infected cells and removing specific segments of HIV's genetic material implanted by the virus. (hivmanagement.net)
After just a couple of weeks, every type of tissue showed signs of the viral genetic material being removed. (hivmanagement.net)
These treatments target diseases with a single, well-characterized genetic cause, meaning the therapy can effectively replace or supplement the function of the faulty gene. (atacana.com)
The stable nature of the site and the ability for scientists to control global or conditional gene expression make the Rosa26 mouse model a versatile genetic tool. (renmab.com)
The ability to follow genes throughout a line allows for a more in-depth study of how genetic code passes down and express themselves over generations. (renmab.com)
For example, one patient might respond well to a therapy targeting tumor blood-vessel growth, while the other patient's tumor might have activated a genetic program that allows it to resist such a therapy. (technologyreview.com)
Then the researchers looked for connections between the ten types of tumors shown in the MRIs and the activity of seven genetic programs by studying the patients' biopsies using microarrays. (technologyreview.com)
These genetic programs included groups of genes associated with blood-vessel growth, cell proliferation, and other characteristic aspects of cancer biology, all of which are targeted by existing drugs. (technologyreview.com)
Human genetic diversity alters off-target outcomes of therapeutic gene editing. (cdc.gov)

As of 2023, we find ourselves in an exciting era for gene therapy, with several therapeutics having received approval and many more under development. (atacana.com)
Under its guiding slogan "The future of medicine will come through mitochondria", the World Mitochondria Society proudly presents Targeting Mitochondria 2023 speakers. (wms-site.com)
Join Targeting Mitochondria 2023 to learn more about Prof. Yamada's interesting talk. (wms-site.com)
Please see below the latest updates regarding the 14th edition of the World Congress on Targeting Mitochondria 2023: Workshop & Conference Progra. (wms-site.com)
Prof. Vladimir Gogvadze, Karolinska Institutet, Sweden, and active member of the scientific committe will join Targeting Mitochondria 2023 t. (wms-site.com)
Prof. Carsten Culmsee, active member of the scientific committe and from the University of Marburg, Germany, will join Targeting Mitochondria 2023 thi. (wms-site.com)
Dr. Egbert Mik, Erasmus MC, The Netherlands, and ative member or WMS scientific committee, will be joining Targeting Mitochondria 2023. (wms-site.com)
Prof. Nils Wiedemann, Universität Freiburg, Germany, will join Targeting Mitochondria 2023 to present a talk entitled: "Mitochondrial Preseq. (wms-site.com)

Scientists have known that the ALK gene frequently mutates in various types of cancer, and it gained a reputation as an oncogene, a gene that drives the development of tumors. (bajaprogofficial.com)
The researchers say that therapeutics targeting the gene might help scientists fight obesity in the future. (bajaprogofficial.com)
In an advance that will help scientists track coronavirus variants in wild and domesticated animals, researchers report they can now detect exposure to the SARS-CoV-2 virus in any animal species. (illinois.edu)
UCLA researchers have combined two tools - gene expression and the use of peripheral blood - to expand scientists' arsenal of methods for pinpointing genes that play a role in autism. (uclahealth.org)
Published in the June 21 online edition of the American Journal of Human Genetics, the findings could help scientists zero in on genes that offer future therapeutic targets for the disorder. (uclahealth.org)
Scientists use human fetal tissue to look at HD brain development. (hdbuzz.net)
By harnessing these viral vectors' ability to efficiently infect both dividing and non-dividing cells, scientists can introduce specific genes into diseased tissues and initiate targeted cellular responses aimed at restoring normal physiological functions. (aliyuncs.com)
The scientists first created a whole-body PCTP knockout male mouse (meaning one in which genes have been activated or deactivated), assessing its fat metabolism under different dietary conditions, including normal and high-fat diet. (anl.gov)
A team of scientists from Stanford University is working with researchers at the Molecular Foundry, a nanoscience user facility located at the Department of Energy's Lawrence Berkeley National Laboratory (Berkeley Lab), to develop a gene-targeting, antiviral agent against COVID-19. (azolifesciences.com)
Then, the 1980s saw the first successful gene transfer into mammalian cells: scientists at MIT and Harvard Medical School successfully transferred bacterial gene encoding resistance to the neomycin analog G418 into pluripotent hematopoietic stem cells using an infectious retrovirus vector 6 . (atacana.com)
In the early 1990s, researchers isolated Rosa26, giving scientists a specific site for inserting genes to study. (renmab.com)
Scientists initially created this line through embryonic stem cell retroviral gene trapping. (renmab.com)
The Rosa26 locus is a useful place for inserting a gene, The location of the insertion is known - not random - and it allows scientists to study a gene without affecting the function of other genes. (renmab.com)

Transcriptomics has also expanded from sampling protein coding genes to non-coding transcripts such as miRNAs, which have the potential not only to act as more stable biomarkers, but also to regulate translation, and in themselves be used a therapeutic agents against specific targets. (videolectures.net)
The PEX7 gene provides instructions for making a protein called peroxisomal biogenesis factor 7, which is part of a group known as the peroxisomal assembly (PEX) proteins. (medlineplus.gov)
It is capable of precisely targeting any type of cell and selectively adding any protein of interest. (duke.edu)
A true mutation will alter a gene's sequence, modifying the protein or RNA it produces - or preventing the gene from producing them entirely. (uclahealth.org)
Its first target will be alpha-1 antitrypsin (AAT) deficiency, a condition caused when a faulty gene fails to produce a protein that protects the lungs from damage when the body's immune system is triggered to fight an infection or by irritants. (imperial.ac.uk)
With gene therapy, we think that we can make enough of the protein in the right compartment, and that will be better than the treatments currently available. (imperial.ac.uk)
Participants in the trial suffer from a condition called hereditary transthyretin amyloidosis, in which a mutated gene produces a malformed protein (transthyretin) that builds up in and damages the heart and nerves. (singularityhub.com)
Blood carried the therapy to the liver, where it switched off the mutated gene and curtailed production of the errant protein. (singularityhub.com)
The cell repairs the DNA at the site of the break, but imperfectly, switching the gene off and shutting down production of the problematic protein. (singularityhub.com)
Existing drugs , approved by the FDA in 2018, silence the mRNA that produces the malformed thyretin protein, instead of altering its gene. (singularityhub.com)
That is, by targeting the genes themselves, the protein is permanently silenced. (singularityhub.com)
The protein is associated with microtubules , and Chatterjee and colleagues silenced the gene via short interfering RNA (siRNA) a molecular biological mechanism to study genes, targeting the CKAP5 encapsulated in lipid nanoparticles for in vivo delivery. (medicalxpress.com)
Cy7 dye is commonly used in fluorescence microscopy, flow cytometry, and in vivo imaging to study various biological processes such as gene expression, cell trafficking, protein localization, and tumor targeting. (axispharm.com)
One of those molecules, phosphatidylcholine transfer protein (PCTP), suppresses the activity of the other, PPARdelta, a gene regulator critical in burning fat and energy metabolism, under conditions of overnutrition. (anl.gov)
Finally, the researchers focused on the specific protein interactions driving the different phenotypes. (anl.gov)
It's not a smoking gun-they would require very sophisticated mass spectrometry or kinetics to look at the transfer of phospholipid from one protein to another-but the researchers were able to support their hypothesis that the interaction between PCTP and PPARdelta is repressive, and when PCTP is unable to bind phosphatidylcholines, it is unable to interact with and repress PPARdelta. (anl.gov)
Researchers at the Francis Crick Institute have found that a protein which is initially helpful in the body's immune response to a virus, can later interfere with the repair of lung tissue. (azolifesciences.com)
Researchers report that smoking increases the gene expression of ACE2, the protein that binds SARS-CoV-2, which may promote COVID-19 infection. (azolifesciences.com)

In particular, methods used to silence genes are being increasingly used to produce therapeutics to combat cancer and other diseases, such as infectious diseases and neurodegenerative disorders. (wikipedia.org)
In addition, attempts are being made to use ribozymes to produce gene silencing therapeutics, which would silence genes that are responsible for causing diseases. (wikipedia.org)
Researchers described technological advances as well as novel miRNA therapeutics such as for cardiovascular disease, cancer, and muscular dystrophy. (genengnews.com)
Researchers at Regulus Therapeutics described how one such miRNA (miR-21) may provide a novel therapeutic target for hepatocellular carcinoma. (genengnews.com)
Following the death of a patient during a gene therapy trial 8 , research focused on improving the safety and efficacy of gene therapeutics before Glybera became the first approved gene therapy in the Western world in 2012, though it was withdrawn from the market in 2017 9 . (atacana.com)

Some medicines targeting the relevant genes are already in use, but for other diseases. (eurekalert.org)
In addition, they provide a more complete view on the development of diseases since diseases are generally associated with genes that have a reduced expression. (wikipedia.org)
This ability has provided an enormously powerful tool for modeling specific diseases when a mutated human gene can be observed in the mouse. (genetargeting.com)
Humanized mouse models are extremely useful for studying complex diseases, such as atherosclerosis and hypertension, as many of the genes responsible for these diseases are shared between mice and humans . (genetargeting.com)
Ion AmpliSeq On-Demand panels provide over 5,000 genes known to be associated with various inherited diseases, including the RASopathies. (thermofisher.com)
Gene therapy has long promised a solution for inherited diseases untreatable with conventional medicine. (imperial.ac.uk)
This cutting-edge technology allows researchers to manipulate the DNA of stem cells with unprecedented precision, leading to breakthroughs in understanding and treating various diseases. (biocellsupply.com)
Additionally, targeting rare diseases with previously unmet medical needs has the advantage of an uncrowded drug market with limited competition. (atacana.com)
Gene therapy holds tremendous promise for treating many diseases, but several challenges remain, including potential immune responses, issues with tissue targeting, and high costs. (atacana.com)

Adipose tissue plays an important role in metabolism through its storage and release of triglycerides, peptide hormones (adipokines) and other proteins, and in the case of brown fat, for its role in thermogenesis ( 1 ). (diabetesjournals.org)
It works by targeting cancer-specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. (bvsalud.org)
The team used a hybrid method that blended tests that read the order of DNA bases with those that analyze gene expression, the process by which genes make cellular proteins. (uclahealth.org)
For example, adenoviral vectors can be used to introduce genes encoding calcium-handling proteins involved in excitation-contraction coupling, which is essential for proper contraction and relaxation of cardiac muscle cells. (aliyuncs.com)
Therefore, this locus provides a useful place for making gene insertions and studying how proteins impact the whole body. (renmab.com)

With a conditional KO, researchers can target specific genes at specific times in a certain tissue type. (cyagen.com)

First identified in embryonic stem cells, SEs mediate pluripotent state maintenance by promoting the expression of pluripotent identity genes, including Oct4 , Sox2 , Nanog and Klf4 . (nature.com)
To generate suitable C57BL/6 embryonic stem cells for gene targeting experiments, the Sanger team established a male cell line (JM8) from the N-substrain of C57BL/6 mice. (umassmed.edu)
A 1997 study of mice grown from embryonic stem cells infected with ROSAβgeo retrovirus showed expression throughout every tissue in the body. (renmab.com)

Senior author Ramin Shiekhattar is a professor in two programs at Wistar, the gene expression and regulation program and molecular and cellular oncogenesis program. (sciencedaily.com)
NIH is launching a program, Molecular Phenotypes of Null Alleles in Cells (MorPhiC), to better understand the function of every human gene and generate a catalog of the molecular and cellular consequences of inactivating each gene. (genome.gov)
With RNA-sequencing now becoming more affordable and with the advantages of identifying novel transcripts and detecting a greater range of expression levels, the future for gene expression profiling includes focusing on transcripts in specific cellular compartments, being an integral part of clinical trials and perhaps even a standard part of diagnostic procedures and therapeutic decisions. (videolectures.net)
By sequencing the messenger RNA molecules in each individual cell - messages that indicate cellular activity - the researchers are able to uncover the molecular makeup of each cell, and to discern a nuanced range of cell identities and functions in a given cell population. (weizmann-usa.org)
By selectively modifying specific genes in stem cells, researchers can investigate the role these genes play in various cellular processes and disease pathways. (biocellsupply.com)
Adenoviral vectors have shown promise in gene therapy for chronic heart failure by targeting key cellular mechanisms involved in the progression of this condition. (aliyuncs.com)

One approach uses modified lentiviruses as vectors to deliver therapeutic genes to the lungs. (imperial.ac.uk)
Adenoviruses are DNA viruses that can be genetically modified to deliver therapeutic genes into target cells, offering a means to correct or replace defective genes responsible for disease pathogenesis. (aliyuncs.com)

A humanized mouse is a mouse that carries functional human genes, cells, tissues, and/or organs. (genetargeting.com)
A multidisciplinary team from two Johns Hopkins University institutions, including neurotoxicologists and virologists from the Bloomberg School of Public Health and infectious disease specialists from the school of medicine, has found that organoids (tiny tissue cultures made from human cells that simulate whole organs) known as "mini-brains" can be infected by the SARS-CoV-2 virus that causes COVID-19. (azolifesciences.com)
that affect activities of target tissues and organs. (msdmanuals.com)

This library would make it easier and faster for researchers to obtain mouse stem cells with specific genes already 'knocked-out' so that they will not need to spend the time. (progress.org.uk)
Gene targeting' uses a piece of DNA to disrupt a gene in a specific place. (progress.org.uk)
In identifying these specific genes, the investigators hope to predict the effect of treatments targeting specific genes. (doctorshealthpress.com)
A new method of targeting specific DNA sequences in zebrafish could accelerate the discovery of gene function and the identification of disease genes in humans. (genome.gov)
However, our strategy allows specific targeting of cancer cells. (sflorg.com)
The results of these studies have been used to investigate disease mechanisms, monitor progression of disease and identify specific genes or gene signatures which could be used as diagnostic or prognostic biomarkers. (videolectures.net)
Researchers are developing drugs that target specific molecular changes. (bvsalud.org)
Researchers said the tool could enable modifying very specific cells and cell functions to manage disease. (duke.edu)
We have the knowledge, not only about the vector platform and the gene therapy background, but also disease-specific knowhow," says Uta Griesenbach , Professor of Molecular Medicine at the National Heart and Lung Institute. (imperial.ac.uk)
While these vectors have a broad tropism for infecting various cell types, non-specific uptake by off-target tissues can lead to unintended side effects. (aliyuncs.com)

Immunodeficient mice (mice without a fully functioning immune system) can be used as hosts to grow both normal and diseased human tissue. (genetargeting.com)
This method has been used to understand everything from the effects of fetal brain development to the way common immune cells function differently in the various tissues of our body, including their involvement in cancer or neurodegeneration. (weizmann-usa.org)
Using mouse models, the researchers will investigate whether succinate signaling alters the oral microbiome, study the role of succinate as an inflammatory and immune mediator, and determine whether blocking succinate signaling can thwart diabetes-related periodontal bone loss. (newswise.com)
Macrophages are specialized immune cells that promote tissue inflammation by secreting inflammatory molecules, which can lead to insulin resistance and metabolic disease. (sciencedaily.com)
The microbes don't invade live tumor cells but live in necrotic tumor tissue, where they apparently avoid clearance by the immune system. (aacrjournals.org)
The immune system has a remarkable capacity to scan tissues to recognize and eliminate abnormal and malignant cells. (lu.se)
We hope to provide the blueprints for a new generation of smart circuit immune cells that can be tailored to perform precise actions upon combinatorial antigen signatures to enable safe and effective targeting of tumor cells. (lu.se)

Composed of multiple constituent enhancers, super enhancers (SEs) are powerful cis-regulatory elements that identify genes that ensure sequential differentiation. (nature.com)

The three universities that make up the UK Respiratory Gene Therapy Consortium (GTC) have developed a gene transfer agent with great promise for the treatment of cystic fibrosis, which has been licensed to the pharmaceutical company Boehringer Ingelheim. (imperial.ac.uk)
After so many years of work, I couldn't be more pleased that one part of our research has been picked up by a big pharmaceutical company, and another has a chance with a spinout, since that represents the best chance for patients to benefit," says Eric Alton , Professor of Gene Therapy and Respiratory Medicine at the National Heart and Lung Institute at Imperial, and coordinator of the GTC. (imperial.ac.uk)
Created in 2001, the GTC brings together researchers from Imperial and the universities of Oxford and Edinburgh to work on gene therapy treatments for cystic fibrosis. (imperial.ac.uk)
This lentiviral vector was the platform technology licensed to Boehringer Ingelheim for further development of cystic fibrosis gene therapy in 2021. (imperial.ac.uk)
The UK Respiratory Gene Therapy Consortium in 2021. (imperial.ac.uk)
Past attempts at gene therapy using adeno-associated virus as a vector have also proved disappointing, and the repeated doses that would be necessary in practice are not possible because the body tends to react to the virus. (imperial.ac.uk)
We believe AlveoGene has potential to become a leader in respiratory gene therapy. (imperial.ac.uk)
Its development has paved the way for advancements in stem cell research , gene therapy , and precision medicine . (biocellsupply.com)
In recent years, the field of regenerative biomedicine has witnessed a paradigm shift with the emergence of gene therapy as a promising treatment modality. (aliyuncs.com)
One particularly influential approach in this regard is the use of adenoviral vectors, which hold great potential for unlocking the therapeutic benefits of gene therapy. (aliyuncs.com)
Gene therapy using adenoviral vectors offers an innovative solution for individuals like John who face debilitating conditions that have traditionally been difficult to manage effectively. (aliyuncs.com)
With further progress in this field, gene therapy using adenoviral vectors may become a transformative approach in improving patient outcomes and quality of life for individuals like John. (aliyuncs.com)
This vision is becoming increasingly plausible with the advent of gene therapy, a promising field that aims to manipulate an individual's genes to prevent or treat disease. (aliyuncs.com)
The Rise of Gene Therapy: Where Are We Today? (atacana.com)
Amid a new era of medicine, gene therapy is a rapidly evolving field at the precipice of revolutionizing healthcare. (atacana.com)
The various types of gene therapy include viral vector-based gene therapy, where a new, exogenous gene is inserted into the body and directly delivered to the required tissue by viral vectors such as adeno-associated virus (AAV) vectors 1 . (atacana.com)
A similar method is non-viral vector-based gene therapy, where synthetic delivery vectors such as lipid- or polymer-based vectors are used for the systemic delivery of DNA 2 . (atacana.com)
However, ASOs are often not considered a type of gene therapy since they act on RNA rather than DNA and therefore only provide a short-lasting, transient therapeutic effect. (atacana.com)
The concept of gene therapy was first proposed in the 1960-70s 5 . (atacana.com)
This was quickly followed by FDA approval of the first human gene therapy trial in 1990 that saw the retroviral-mediated transfer of functional adenosine deaminase (ADA) into the T-cells of severe combined immunodeficiency (SCID) patients 5,7 . (atacana.com)
Gene Therapy: Where Are We Now? (atacana.com)
Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. (cdc.gov)
This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy. (cdc.gov)

Bone-targeting ZBTB16 overexpression had a therapeutic effect on the decreased bone density and remodeling capacity of Brd4 fl/fl Prx1 -cre mice and osteoporosis (OP) models. (nature.com)
Recently, researchers from the Sanger Institute (UK) have isolated stable and germline competent embryonic stem (ES) cells from C57BL/6N mice (1). (umassmed.edu)
When these C57BL/6N-based JM8 cells were injected into blastocysts from albino mice, a high proportion of chimeras displayed ES cell contribution to both germline and somatic tissues. (umassmed.edu)
The Sanger group performed gene targeting in the JM8 ES cells to delete the retrotransposon from the agouti locus and restore agouti gene function, permitting the visualization of ES cell-derived mice on an inbred C57BL/6 background by agouti coat color. (umassmed.edu)
They show that deleting this gene results in thinner flies and mice and find that expression of it in the brain may be involved in regulating energy expenditure. (bajaprogofficial.com)
The researchers also found that flies and mice without ALK remained thin and were resistant to diet-induced obesity. (bajaprogofficial.com)
Treating obese mice with catestatin (CST), a peptide naturally occurring in the body, showed significant improvement in glucose and insulin tolerance and reduced body weight, report researchers. (sciencedaily.com)
The researchers engineered nonpathogenic strains of Escherichia coli , Bifidobacterium breve , and Salmonella typhimurium to express the bioluminescence gene lux and injected them intravenously into mice with various solid tumors. (aacrjournals.org)
Researchers separated the mice that had the Rosa26 insertion for future study. (renmab.com)
Prior to gene targeting, researchers created transgenic mice. (renmab.com)
Study Homozygotes Resulting From Knock-In While mice with combined genotypes prove useful for study, mice with singular genotypes from knocking in a gene at the Rosa26 site are also beneficial. (renmab.com)
The viability of these mice ensures the longer study of the results of the added gene at the Rosa26 location. (renmab.com)
This team showed that injection of just three genes- Neurog3, Mafa, and Pdx1 -into the pancreatic parenchyma of mice leads to conversion of exocrine cells to functional beta cells, a process popularly known as "reprogramming. (medscape.com)

Conditional gene targeting has been extensively used for in vivo analysis of gene function in adipocyte cell biology but often with debate over the tissue specificity and the efficacy of inactivation. (diabetesjournals.org)
Additionally, we've made progress in hepatocyte-targeted in vivo gene silencing by modifying the siRNAs. (genengnews.com)

Cell cultures have played a critical role in modern infectious disease research, particularly in the area of viruses and the expression of viral gene products," Bloom says. (the-scientist.com)
Gene silencing is the regulation of gene expression in a cell to prevent the expression of a certain gene. (wikipedia.org)
When genes are silenced, their expression is reduced. (wikipedia.org)
The antisense oligonucleotides can affect gene expression in two ways: by using an RNase H-dependent mechanism or by using a steric blocking mechanism. (wikipedia.org)
Ribozymes are catalytic RNA molecules used to inhibit gene expression. (wikipedia.org)
RNA interference (RNAi) is a natural process used by cells to regulate gene expression. (wikipedia.org)
Called a deacetylase, this enzyme removes acetyl groups from histones to repress gene expression. (sciencedaily.com)
In the body's scheme for safely storing genes away until needed, DNA is tightly looped around the histones, kept secure by enzymes similar to the ones studied by the Wistar team until made accessible by the activity of other enzymes responsible for gene expression. (sciencedaily.com)
The involvement of miRNAs in cancer research emerged from studies demonstrating their aberrant expression in neoplastic tissues. (genengnews.com)
Gene expression profiling has come a long way since the term was first widely used in the mid to late 1990's. (videolectures.net)
With the advent of microarrays allowing the expression levels of increasing numbers of genes to be quantified, gene expression profiling, microarray analysis and transcriptomics have become frequently used terms associated with sampling RNA from multiple different tissue types. (videolectures.net)
Gene expression profiling has been a key methodology used in Sheffield and we initially used it to investigate disease mechanisms associated with mutant SOD1. (videolectures.net)
The Cre/lox system has become the cornerstone of modern mouse genetics because it enables sophisticated control over the timing and location of gene expression. (jax.org)
Using a knock-in Cre also allows you to evaluate the likelihood of any unexpected expression by first reviewing the expression patterns of the endogenous gene. (jax.org)
Therefore, our study shows that SEs orchestrate the osteogenesis of MSCs by targeting ZBTB16 expression, which provides an attractive focus and therapeutic target for osteoporosis. (nature.com)
Dysregulation of SE-mediated ZBTB16 expression leads to osteoporosis, and bone-targeting ZBTB16 overexpression is efficient in accelerating bone repair and treating osteoporosis. (nature.com)
The non-agouti mutation in C57BL/6 strains is due to an 11.8 Kbp retrotransposon located in the first intron of the agouti gene that abolishes the expression of the Agouti gene. (umassmed.edu)
Monitoring gene expression provides us with another line of data to inform our understanding of how autism develops," said Geschwind, who also directs the Center for Autism Research and Treatment at the Semel Institute for Neuroscience and Behavior at UCLA. (uclahealth.org)
We found that we can use gene expression to help understand whether a rare mutation is causing disease or playing a role in disease development," said Geschwind. (uclahealth.org)
Brain cells and genes related to synaptic function are expressed in the blood, offering a window into gene expression. (uclahealth.org)
Unlike most other genes, expression levels of many of the 50 mutation-containing genes that form the suspected network were highest early in fetal development, tapered off by childhood, but conspicuously increased again in early adulthood - just when schizophrenia symptoms typically first develop. (neurosciencenews.com)
The implicated genes play important roles in migration of cells in the developing brain, communication between brain cells, regulation of gene expression, and related intracellular workings. (neurosciencenews.com)
Gene expression analysis suggested that the differences derived from repression of PPARdelta gene regulation activity. (anl.gov)
The high level of expression of genes inserted into the Rosa26 site makes it desirable for researchers. (renmab.com)
Researchers at the University of California, San Diego, have made connections between features in magnetic resonance images of the most prevalent form of brain cancer (above) and gene-expression patterns. (technologyreview.com)
Gene-expression patterns result in anatomical changes you can see in these images," says Webster Cavanee , director of the Ludwig Institute for Cancer Research at the University of California, San Diego. (technologyreview.com)
Last year, Kuo published a paper connecting characteristics of liver tumors in CT scans with gene-expression patterns. (technologyreview.com)
This suggests that the connection between anatomical changes and gene expression will hold up across imaging and tumor types alike. (technologyreview.com)

Technologies for obtaining conditional knockouts are also developed and offer researchers more flexibility to obtain desired models. (cyagen.com)
TurboKnockout ® , Cyagen's proprietary and the fastest ES-cell based gene targeting technology, comparing to traditional gene targeting technology, can eliminate two generations of breeding, allowing generation of conditional knockout mouse models in as fast as 6 months. (cyagen.com)

The process to silence genes first begins with the entrance of a double-stranded RNA (dsRNA) molecule into the cell, which triggers the RNAi pathway. (wikipedia.org)
When a direct replacement for the deleted metabolic gene is not available, our algorithms use a mathematical model of the cancer cells' metabolism to predict the paralogous metabolic pathway they might use," said Abhinav Achreja, a U-M research fellow in biomedical engineering and lead author on the research paper. (sflorg.com)
Several groups, including ourselves have now identified drugs targeting this pathway and it remains a viable target for therapeutic intervention. (videolectures.net)
Our results implicate networked genes underlying a pathway responsible for orchestrating neurogenesis in the prefrontal cortex in schizophrenia. (neurosciencenews.com)

As beneficial as these treatments are, researchers are still looking into finding an actual cure for the disease. (hivmanagement.net)
This advance could help neurosurgeons make critical decisions about the extent of tissue removal and possible on-the-spot treatments. (neurosciencenews.com)
Reviews in Cell Biology and Molecular Medicine articles are self-contained treatments of a given topic, usually with over 100 references and 10 to 20 figures and tables, making this an authoritative reference source for undergraduate and graduate students, faculty, librarians and researchers. (lu.se)

The technology, which earned two of its discovers a Nobel Prize in 2020, can target and edit genes more easily than its predecessors. (singularityhub.com)

DURHAM, NC - Duke University researchers have developed an RNA-based editing tool that targets individual cells, rather than genes. (duke.edu)
The group envisioned targeting many genes at once, including combined targets in the same cell, and then identifying the resulting changes to the cell and its function. (weizmann-usa.org)
To overcome these obstacles in the translation of novel findings to clinical applications, researchers must address the most common targets and upstream mechanisms mediating the key steps of MSC osteogenesis. (nature.com)
Knowing this mechanism of action can help researchers and physicians better understand the illness and provide new treatment targets. (illinois.edu)
The researchers used the combined method to prioritize gene targets that merit closer investigation. (uclahealth.org)

A new study shows that an enzyme involved in appropriately repressing sets of neuronal genes in non-neuronal cells is also a target for certain monoamine oxidase inhibitors used to treat depression. (sciencedaily.com)
In 2005, professor Ramin Shiekhattar, Ph.D., at The Wistar Institute and his colleagues reported details about an enzyme involved in appropriately repressing sets of neuronal genes in non-neuronal cells. (sciencedaily.com)
This week's neuroscience revelations encompass intriguing findings from the enigmatic genetics of mind-controlling hairworms to the groundbreaking link between alcohol use disorders and certain neuronal plasticity genes. (neurosciencenews.com)

Researchers also use cells as biological test tubes to grow viral stocks. (the-scientist.com)
Reverse transcription polymerase chain reaction (RT-PCR) was used to detect viral RNA in brain tissue. (who.int)

Instead, this substance gradually builds up in the body's tissues. (medlineplus.gov)

The latter is the same ribonucleoprotein machinery associated with siRNA-mediated gene silencing. (genengnews.com)
In the case of a GalNAc-conjugated siRNA targeting transthyretin (TTR), target gene silencing was achieved with an ED50 of approximately 5 mg/kg with a single subcutaneous injection. (genengnews.com)
These results represent a greater than 30-fold improvement in target gene silencing with siRNA conjugates as compared with first-generation siRNA conjugates previously described. (genengnews.com)

Within the field of ALS, researchers have moved from using pooled spinal cord homogenates, to ensure they had sufficient material, through to isolating individual cell types from post-mortem cases and utilising peripheral tissues to determine biomarkers of disease and progression or stratification of patients. (videolectures.net)
Cancer surgeons may soon have a more complete view of tumors during surgery thanks to new imaging agents that can illuminate multiple biomarkers at once, University of Illinois Urbana-Champaign researchers report. (illinois.edu)

Methods using gene silencing are often considered better than gene knockouts[citation needed] since they allow researchers to study essential genes that are required for the animal models to survive and cannot be removed. (wikipedia.org)
The approach using multiple methods including targeted Next-Generation Sequencing (NGS) is an appealing option for researchers since targeted NGS can accurately interrogate many genes in one test which reduces cost and speeds time. (thermofisher.com)

Researchers at the Institute of Molecular Pathology and Immunology of the University of Porto (IPATIMUP) has been developing targeted NGS assays to further clinical research into Noonan syndrome. (thermofisher.com)

EUCOMM is forecasted to produce 20,000 ' mutant ' mouse stem cells , each with one gene 'knocked-out', and will complement the World Stem Cell Hub. (progress.org.uk)
A team of South Korean researchers have managed to extract embryonic stem (ES) cells from frozen human embryos. (progress.org.uk)
The Adipoq-Cre exhibited no observable recombination in any other tissues examined, whereas both aP2-Cre lines resulted in recombination in endothelial cells of the heart and nonendothelial, nonmyocyte cells in the skeletal muscle. (diabetesjournals.org)
After analyzing the cells of more than 100 tumors, including 25 ovarian cancer tumors, the team of researchers reported that identifying genes like PAX8 might help in the battle against certain types of cancer. (doctorshealthpress.com)
It means the researchers are looking for genes that, when inactivated, affect the survival of cancer cells. (doctorshealthpress.com)
genes in an attempt to find those needed for cancer cells to grow and survive. (doctorshealthpress.com)
Meanwhile, at the DMR level, the researchers found more than 10,000 hypo-DMRs between the two esophageal cancer types and also note that many of the methylation changes there were also present in normal cells. (genomeweb.com)
A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy. (genomeweb.com)
The obvious place to start is with cells that match the animal and tissue that naturally host the virus. (the-scientist.com)
The way that tumor cells enable their uncontrolled growth is also a weakness that can be harnessed to treat cancer, researchers at the University of Michigan and Indiana University have shown. (sflorg.com)
Their machine-learning algorithm can identify backup genes that only tumor cells are using so that drugs can target cancer precisely. (sflorg.com)
Most cancer drugs affect normal tissues and cells. (sflorg.com)
This could revolutionize the precision medicine field because the drug targeting will only affect and kill cancer cells and spare the normal cells," said Deepak Nagrath, a U-M associate professor of biomedical engineering and senior author of the study in Nature Metabolism . (sflorg.com)
This approach is known as collateral lethality-using information gleaned from genes that cancer cells discard to find weaknesses. (sflorg.com)
Cancer cells themselves used to contain suppressor genes that prevent their spread. (sflorg.com)
In doing so, the cells typically lose other genes that are necessary for survival. (sflorg.com)
To avoid death, the cells find a paralog-a gene that can serve a similar function. (sflorg.com)
In examining ovarian cancer cells, U-M's team zeroed in on one gene, UQCR11, that was often deleted along with a suppressor gene. (sflorg.com)
The main gene finding, ADRA2A , "points to a receptor on the cells that regulate the tone of these blood vessels," she continued. (medscape.com)
Tagging and illuminating only the inhibitory "brake" cells (green) in human brain tissue is just one of many things the new tool, CellREADR, can do. (duke.edu)
The new profiling technology enables researchers to manipulate gene functions within single cells, and understand the results of each change in extremely high resolution. (weizmann-usa.org)
Thousands of cells from a particular tissue in the body can be sequenced at once in his lab, uncovering variation in cell identities and functions. (weizmann-usa.org)
The UMass Chan TAMC has routinely utilized a subclone (MK6) of 129 SVevBrd derived ES cells in gene-targeting experiments. (umassmed.edu)
The UMass Chan Transgenic Animal Modeling Core (TAMC) also has JM8-strain ES cells for targeting, and routinely injects either JM8.F6-strain ES cells or JM8.A3-strain ES cells into albino C57BL/6J Tyrc-Brd blastocysts. (umassmed.edu)
Researchers have taken connective tissue cells that have been mechanically reprogrammed to resemble stem cells and transplanted them into damaged skin. (nanowerk.com)
Researchers developed a new program that provides a more accurate understanding of the peptide sequences in cells. (nanowerk.com)
These measurements can help understand how cells and tissues survive under pressure, and reveal how problems in regulating pressures lead to disease. (nanowerk.com)
One challenge that stands in the way is developing a gene transfer agent that will deliver a normal copy of the gene in question to the cells in the body where it is needed, and effectively set it to work. (imperial.ac.uk)
In the July 6 issue of Cell Stem Cell , researchers at the University of California, San Diego School of Medicine describe how human epidermal progenitor cells and stem cells control transcription factors to avoid premature differentiation, preserving their ability to produce new skin cells throughout life. (scitechdaily.com)
Without a functioning exosome in progenitor cells," said Sen, "the progenitor cells prematurely differentiate due to increased levels of GRHL3 resulting in loss of epidermal tissue over time. (scitechdaily.com)
The most recent trial included targeting HIV material in organ tissue and blood cells. (hivmanagement.net)
This new approach allows the fine-tuning of gene activity in human cells. (neurosciencenews.com)
For many years, researchers have analyzed the use of liposomes, which are hollow spheres composed of lipid bilayers, to transmit chemotherapy drugs to tumor cells. (azolifesciences.com)
This disruptive realm of medicine involves genetically modifying cells by introducing, altering, or suppressing a gene to produce a desired therapeutic effect. (atacana.com)
For example, how can genes be delivered at the precise dose to cells in humans? (medscape.com)
[ 7 , 8 ] As background, in 2009, Collombat and colleagues [ 9 ] showed that pancreatic misexpression of the gene Pax4 led to the conversion of glucagon -producing alpha cells into functional beta cells. (medscape.com)

But in the latest bit of health news on the cancer front, researchers at MIT and Harvard have found that a particular gene may be altered in ovarian tumors. (doctorshealthpress.com)
The PAX8 gene was identified in nearly one-fifth of the ovarian tumors analyzed. (doctorshealthpress.com)
This dramatic observation led the researchers to undertake the current study to test if modulating Atoh1 levels in the cerebellar neurons would provide a better molecular handle to treat Shh-type medulloblastoma tumors. (texaschildrens.org)
The San Diego researchers defined a longer list of characteristics, including morphology and the interaction of the tumors with the surrounding tissues. (technologyreview.com)

To our knowledge, this is the first demonstration of the prominent cell-type specificity of PMDs across normal, precursor, and malignant states," the researchers write. (genomeweb.com)
The tool's remarkable tissue specificity relies on the fact that each cell type manufactures signature RNA not seen in other cell types. (duke.edu)

These molecules work by cleaving mRNA molecules, essentially silencing the genes that produced them. (wikipedia.org)
The enzyme's function is to remove methyl groups from small molecules called histones to modify them in ways that trigger gene repression. (sciencedaily.com)
Using the Advanced Photon Source (APS), a U.S. Department of Energy (DOE) Office of Science user facility at DOE's Argonne National Laboratory, a team of researchers has uncovered an interaction between two molecules that may repress fat-burning under high-fat diet. (anl.gov)
Are there ways to mimic the effects of genes with hormones or small molecules? (medscape.com)
With the exception of chloroform, the rest of trihalomethanes (THM), the so-called brominated THM (Br-THM) are composed of bromine-substituted molecules with increased lipophilicity and potency to biological tissues. (who.int)

Furthermore, more literatures suggest gene-environment interaction as a potential contributor to disease mechanism. (cdc.gov)
This would allow them to focus on mechanism while defraying the possibility that the knockout effect could be acting through other tissue, such as muscle or heart. (anl.gov)

A knock in Cre is a type of mutation where the Cre coding sequence either replaces the coding sequence of an endogenous gene (for example, B6.129P2(C)-Cd19 tm1(cre)Cgn /J ) or is appended to the end of the RNA message using an "internal ribosomal entry site," which produces a bicistronic message encoding both the endogenous gene and Cre. (jax.org)

The outcomes highlighted the importance of the gene of interest as a therapeutic target to investigate genetically unstable ovarian cancers to further elucidate its mechanisms of action. (medicalxpress.com)

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants. (genomeweb.com)
King and colleagues set out to explore causes of schizophrenia by integrating genomic data with newly available online transcriptome resources that show where in the brain and when in development genes turn on. (neurosciencenews.com)
Researchers hope that MRI will soon be used for dividing glioblastomas into genomic subtypes, in turn allowing doctors to put patients on the best drug before a biopsy is even taken. (technologyreview.com)

In a study published in Diabetes on February 6, researchers identified CST's role in the recruitment and function of macrophages in the liver as well as regulation of obesity-induced liver inflammation and insulin resistance. (sciencedaily.com)

A team of researchers set out to determine that with a new comprehensive study in pre-manifest HD young adults. (hdbuzz.net)

Now researchers have wrapped up the largest study ever conducted in the entire world. (eurekalert.org)
Fully 155 researchers from nine different countries collaborated on the study. (eurekalert.org)
Furthermore, our in vitro study indicated that the epithelial-to-mesenchymal transition (EMT), a pre-process of tissue fibrosis, was induced by an mTOR inhibitor in lung alveolar epithelial cell lines carrying the G allele homozygous genotype which was associated with a higher risk of ILD. (bvsalud.org)
In a secondary analysis, the team also used health records from the Queen Mary University of London Genes & Health Study, which contains health information on individuals of South Asian ancestry. (medscape.com)
Brain tissue from people with autism is not readily available for study, and some people are reluctant to use non-neural tissue in psychiatric disease," Geschwind explained. (uclahealth.org)
Malaysia has the highest prevalence of diabetes among all Asian countries, making Malaysians particularly vulnerable to the development of learning and memory deficits," says Nurdiana Samsulrizal, UiTM researcher and first author of the study published in the Journal of Traditional and Complementary Medicine. (issuu.com)
In this study, they show that PCTPs can repress the activity of a nuclear receptor called PPARdelta, a gene regulator critical in burning fat, maintaining energy balance, and regulating fatty acid uptake. (anl.gov)
Knocking out genes is one method used to study how the absence of certain genes will affect an organism. (renmab.com)
Such a study becomes especially useful in identifying gene function through its absence. (renmab.com)

Profundice en los temas de investigación de 'Molecular survey of Bartonella spp. (unab.cl)

Both miRNAs and siRNAs need to be delivered into the target tissue or cell in order to activate the desired therapeutic effect," Muthiah Manoharan, Ph.D., senior vp, drug discovery, Alnylam Pharmaceuticals, explained. (genengnews.com)

The team also looked for associations between tumor appearance and overexpression of one gene in particular, coding for epidermal growth factor receptor, a cell receptor that's overactive in many glioblastomas. (technologyreview.com)

Current treatment options are limited to surgical removal of the tumor and surrounding healthy brain tissue in combination with chemotherapy and radiation. (texaschildrens.org)

orthogonal exPeriMental changes and pathways induced by EGE investigated the impact of aSSeSSMent of ePigenetic environmental factors in cancer causation, both current H. pylori infection and driver geneS and their link to underpinning studies of etiology e , pigenetic memory of past (eradicated) environMental carcinogenS carcinogen evaluation, and prevention. (who.int)

Streptococcal pathogens continue to evade concerted efforts to decipher clear-cut virulence mechanisms, although numerous genes have been implicated in pathogenesis. (cdc.gov)

It also worked well in rats, and in human brain tissue collected from epilepsy surgeries. (duke.edu)
The team's mouse studies also suggested that ALK, which is highly expressed in the brain, plays a part there by instructing the fat tissues to burn more fat from food. (bajaprogofficial.com)
Because HD causes a loss of neurons in the brain, some researchers are exploring ways to replace them. (hdbuzz.net)

But one limitation for replicating these findings is that biobanks that collect biological or medical data and tissue samples don't have a universal standard in data collection, which makes comparability a challenge. (bajaprogofficial.com)
Newswise - The National Institute of Dental & Craniofacial Research, part of the National Institutes of Health, has awarded a grant to researchers at New York University College of Dentistry (NYU Dentistry) to explore the biological mechanisms that contribute to poor oral health and related bone loss among people with diabetes. (newswise.com)
The National ALS Biorepository collects, processes, stores, and distributes a variety of biological specimens such as blood, urine, and tissue from a sample of persons with ALS enrolled in the National ALS Registry who agree to take part in the Biorepository. (cdc.gov)
Its excitation and emission spectra lie in the range of 750-800 nm, which is ideal for imaging applications due to the low tissue autofluorescence and high penetration depth of NIR light in biological tissue. (axispharm.com)

Researchers are confident that if used alone, it has the potential to eliminate HIV from the body. (hivmanagement.net)

Researchers have identified two genes that may contribute to Raynaud phenomenon, a condition where blood vessels in the extremities constrict and limit blood flow. (medscape.com)
Less is known about this gene, but the data we do have suggest that it is involved with regulating the dilation of blood vessels, Pietzner noted. (medscape.com)
One potential application is the delivery of genes encoding growth factors or cytokines that promote angiogenesis, the formation of new blood vessels. (aliyuncs.com)

The researchers, based at the Maria Infertility Hospital in Seoul, have obtained seven ES cell lines from 20 embryos left over from infertility treatment. (progress.org.uk)
Usually there are one or, possibly, two genes that can step in and perform the same function to keep the cell alive. (sflorg.com)
U-M's algorithm correctly sorted through multiple options and successfully predicted a cell missing UQCR11 would turn to the gene MTHFD2 as its backup supplier of NAD+. (sflorg.com)
In recent years, this bacterial system has been adopted by researchers to snip out or insert nearly any gene in any organism or cell, quickly and efficiently. (weizmann-usa.org)
But single-cell sequencing has mostly been an observatory tool, providing a snapshot of a given tissue sample. (weizmann-usa.org)
The researchers screened 20 solid cancer cell lines relative to gene silencing to identify a highly responsive chemo-resistant ovarian cancer cell line that underwent significant depletion in mitotic spindle-dynamics for effective experimental cancer treatment. (medicalxpress.com)
However, existing mitosis-targeting chemotherapeutic agents do not discriminate between healthy and malignant cell lines, resulting in severe side-effects . (medicalxpress.com)
It encodes a nonessential RNA that replicates throughout the body and every cell/tissue in the body that expresses it. (renmab.com)
By adding a reporter gene to this locus, researchers can trace cell lineages. (renmab.com)

Human organoids are small, self-organized, three-dimensional (3D) tissue cultures that have started to revolutionize medical science in terms of understanding disease, testing pharmacologically active compounds, and offering novel ways to treat disease. (mdpi.com)

Its stability, high quantum yield, and low toxicity make it a popular choice for many researchers and medical professionals. (axispharm.com)
Through serendipity, researchers at the University of Pittsburgh Graduate School of Public Health considerably reduced the toxicity of a potential antibiotic against the most feared drug-resistant bacteria, while also improving its stability in fighting infections. (azolifesciences.com)

Anatomy3

Organisms1

Diseases2

Chemicals and Drugs3

Analytical, Diagnostic and Therapeutic Techniques and Equipment35

Psychiatry and Psychology25

Phenomena and Processes9

Disciplines and Occupations37

Anthropology, Education, Sociology and Social Phenomena39

Technology, Industry, Agriculture3

Humanities19

Information Science67

Named Groups6

Health Care90

Geographicals8

Therapies23

CRISPR17

Mutations21

Genome16

Receptors3

Promoters3

Genetic20

20238

Scientists13

Protein18

Therapeutics5

Diseases9

Proteins5

Target specific genes1

Embryonic stem3

Cellular6

Deliver therapeutic genes2

Organs3

Specific10

Immune7

Identify genes1

Therapy23

Mice13

Vivo2

Expression28

Conditional2

Pathway4

Treatments3

20201

Targets5

Neuronal3

Viral2

Body's1

SiRNA3

Biomarkers2

Methods2

Clinical1

Cells36

Tumors4

Specificity2

Molecules5

Mechanism2

Endogenous gene1

Genetically1

Genomic3

Regulation1

Team of researchers1

Study9

Molecular1

Therapeutic effect1

Overexpression1

Chemotherapy1

Infection1

Pathogenesis1

Brain3

Biological4

Eliminate1

Blood vessels3

Cell9

Organoids1

Toxicity2