Cell gene. Medical search. Frequent questions

This segment is required for high-level beta-globin gene expression but gets clipped out during the normal vector preparation process if it is left in the natural, forward direction. (nih.gov)
The two gene therapy trials recently approved for SCD use different ways to change the expression of an individual's genes but both therapies result in an individual making more fetal hemoglobin, a type of oxygen-carrying blood protein present at birth. (nih.gov)
In addition, the medicine that is needed to allow for the new gene expression can have serious side effects. (nih.gov)
Insulin gene expression involves a wide variety of transcription factors, some of which interact with one another [ 25 ] . (medscape.com)
Gene regulation can occur at any point during gene expression, but most commonly occurs at the level of transcription (when the information in a gene's DNA is passed to mRNA). (medlineplus.gov)
The Genetic Science Learning Center at the University of Utah offers an explanation of gene expression as it relates to disease risk . (medlineplus.gov)
Additional information about gene expression is available from yourgenome.org, a service of the Wellcome Trust. (medlineplus.gov)
Dillman AA, Majounie E, Ding J, Gibbs JR, Hernandez D, Arepalli S, Traynor BJ, Singleton AB, Galter D, Cookson MR. Transcriptomic profiling of the human brain reveals that altered synaptic gene expression is associated with chronological aging.Sci Rep. 2017 Dec 4;7(1):16890. (nih.gov)
Abstract: DESCRIPTION (provided by applicant): This application is for partial support for the FASEB Summer Conference on "Nutrient Control of Gene Expression & Cell Signaling. (nih.gov)
The goal of this conference is to bring together scientists from many different disciplines interested in the regulation of gene expression and cell signaling by nutrient and non-nutrient dietary factors. (nih.gov)
The initial sessions of the meeting are organized around the central themes of molecular regulation of gene expression by nutrients, such as glucose and fatty acids, by vitamins and minerals, and by phytochemicals and xenobiotics. (nih.gov)
The method simultaneously measures the expression of multiple genes, allowing scientists to quickly characterize cells according to their function and stage of development. (nih.gov)
They also use a technique called quantitative RT-PCR to measure the expression of genes that indicate ongoing cell development and function. (nih.gov)
For example, expression of the gene SOX2 is much higher in iPS cells than mature RPE. (nih.gov)
Starting with cells from a skin biopsy, the researchers generated iPS-derived RPE and then measured the expression of eight genes that are markers of development, function, and disease. (nih.gov)
The researchers then used the multiplex assay to compare gene expression of iPS-derived RPE to iPS cells and to RPE cells cultured from an eye. (nih.gov)
This report demonstrates proof of principle that relevant gene expression changes in iPS-derived RPE can be measured with confidence using a fully automated, high-throughput assay," said Dr. Bharti. (nih.gov)
Over the past few years, gene expression profiling has been suggested as the best or only way of determining ex vivo drug sensitivity. (cancer.org)
CCDRTs can actually integrate all the gene expression into one convenient test result. (cancer.org)
This initiative encourages research to develop new, or adapt existing, computational and statistical approaches to aggregate, mine, and conduct integrative analyses of single-cell and bulk tissue multi-omic datasets from brain samples (e.g., gene expression, chromatin state, histone modifications, DNA methylation, etc.) to achieve new insights into the biology of psychiatric disorders. (nih.gov)
The discovery of 'clock genes' also led to the realization that the capacity for circadian gene expression is widespread throughout the body. (nih.gov)
We have used tissue-specific, conditional gene expression methods to analyze the relative contributions of central and peripheral circadian oscillators to circadian organization. (nih.gov)
Using cancer cell samples from lymphoma patients, Dr. Teitell and his colleagues discovered that abnormal TCL1 expression is a frequent abnormality in B-NHL. (lymphomainfo.net)
Max Planck researcher Dominic Grün now presents a method based on single-cell data to quantify this variability in gene expression. (mpg.de)
The advantage of the so-called VarID method is that the noise of gene expression can be measured across groups of very similar or related cell states. (mpg.de)
Accordingly, differences in the expression of such genes are difficult to detect in the data. (mpg.de)
Moreover, currently available analysis methods are almost exclusively focused on quantifying and interpreting gene expression levels within an individual cell. (mpg.de)
But the biological implications of gene expression noise during cell differentiation and cell state transitions have not been explored in depths," says Grün. (mpg.de)
VarID connects cells based on the similarity of their gene expression profiles and quantifies gene expression variability - or noise - in local groups of similar cells. (mpg.de)
The new VarID method by Dominic Grün addresses this gap by quantifying the noise of gene expression across groups of very similar or related cell states. (mpg.de)
With this approach, it is possible to explore the dynamics of gene expression noise during the differentiation of stem cells into mature cell types and to investigate the extent to which noise regulates development or is even necessary for cellular differentiation. (mpg.de)
The core of the VarID method is an algorithm developed by Dominic Grün that quantifies the dynamics of gene expression variability from single-cell RNA-sequencing data. (mpg.de)
Thus, VarID delineates neighborhoods with differential gene expression variability, also in complex mixtures of different cell types or cell states. (mpg.de)
Using a gene expression profiles between these two DPC groups, we identified a new transcript factor, distal-less homeobox 4 (DLX4), which was highly expressed in immature teeth DPCs and significantly promoted human iPSC generation in combination with OCT3/4, SOX2 and KLF4. (nature.com)
Knocking down NFATc2 expression, eliminating NFAT DNA binding sites, or interfering with NFAT nuclear import prevented association of MAPKs with gene promoters. (nih.gov)
Inhibiting p38 and JNK activity increased NFAT-ERK association with promoters, which repressed TNF-α and enhanced insulin gene expression. (nih.gov)
Histone acetyltransferase/HDAC exchange was reversed on the insulin gene by p38/JNK inhibition in the presence of glucagon-like peptide 1, which enhanced gene expression. (nih.gov)
To carry out these behaviors, each cancer cell uses its genes to construct the needed molecules in a unique way known as its 'gene expression signature. (cedars-sinai.edu)
The first enabled the researchers to read the gene expression signatures of individual cancer cells from patients with leukemia. (cedars-sinai.edu)
Using this experimental system, the team analyzed the gene expression signatures of a representative sample of barcoded leukemia cells, and then transplanted the remainder of the cells into mice. (cedars-sinai.edu)
Distinct gene expression signatures correlated with the various organs where the cancer cells ended up in the mice. (cedars-sinai.edu)
For example, cancer cells with a high expression of one type of gene tended to colonize the ovaries, while cells with low levels of a different gene expression established colonies in the blood and spleen. (cedars-sinai.edu)
By demonstrating that cancer cells with distinct gene expression signatures tend to grow in different organs and bone marrow pockets, the study also underscored a major problem facing cancer researchers: studying non-representative samples of patient cells. (cedars-sinai.edu)
The new findings demonstrate that serial transplantations also favor the survival of cancer cells with certain gene expression signatures. (cedars-sinai.edu)
Distinct gene expression signatures emerged in leukemia cells that eventually died from intensive treatment, and ceased growing due to maintenance therapy. (cedars-sinai.edu)
When genes get amplified, it looks like they are scooping up regulatory elements that are controlling their expression," Dr. Scacheri said. (cancer.gov)
When the scientists used DNA editing techniques to interfere with each of the switches individually, EGFR gene expression and cell survival dropped. (cancer.gov)
It's possible that, in DNA circles, contacts between genes and enhancers that normally would have been prevented in the chromosome are driving cancer cell growth via additional boosts to gene expression," Dr. Fingerman noted. (cancer.gov)
RT-qPCR was applied to determine mRNA of m6A-associated genes and SFRP2, and western blot were used for ZEB1 and MMP9 protein expression. (nature.com)
Exosome-transmitted lncRNA UFC1 promotes non-small-cell lung cancer progression by EZH2-mediated epigenetic silencing of PTEN expression. (nature.com)
Roundtree IA, Evans ME, Pan T, He C. Dynamic RNA modifications in gene expression regulation. (nature.com)
GXD's primary emphasis is on endogenous gene expression during development. (jax.org)
In this work, 119 overlapping differentially expressed genes (DEGs) were obtained from GSE5281 and GSE122063 datasets through differential expression analysis. (hindawi.com)
A new generation of high-throughput sequencing technologies and the development of genomics have produced a wealth of disease gene expression data and clinical information already stored in many public databases [ 16 - 18 ]. (hindawi.com)
In this study, we used high-throughput sequencing data for differential gene expression analysis, GO functional and KEGG pathway enrichment analyses, and protein-protein interaction (PPI) network analysis to identify network hub genes and their biological roles. (hindawi.com)
AD gene expression data were obtained from Gene Expression Omnibus (GEO) database [ 19 ] ( https://www.ncbi.nlm.nih.gov/gds ). (hindawi.com)
Firstly, the gene expression matrices of GSE5281 and GSE122063 datasets were normalized and formatted into input file format of R language. (hindawi.com)
Researchers describe how gene expression in different human brain regions is altered with age. (the-scientist.com)
To learn what happens within the aging brain and potentially gain information relevant to human health, researchers examined gene-expression patterns in postmortem brain samples. (the-scientist.com)
Overall, the researchers found, gene expression of glial cells changed more with age than did that of neurons. (the-scientist.com)
These gene-expression changes were most significant in the hippocampus and substantia nigra, regions damaged in Alzheimer's and Parkinson's diseases, respectively, according to the study published today (January 10) in Cell Reports . (the-scientist.com)
The authors' effort in this comprehensive work is a 'genomic tour de force,' showing that, overall, non-neuronal cells undergo gene expression changes at a larger scale than previously thought in aging," Andras Lakatos , a neuroscientist at the University of Cambridge, U.K., who was not involved in the study, wrote in an email. (the-scientist.com)
According to the authors, the present study, which used array-based gene-expression data and tissue samples from the UK Brain Expression Consortium and the North American Gene Expression Consortium, represents the largest analysis of aging and gene expression in the human brain. (the-scientist.com)
The researchers examined gene-expression data from 10 brain regions of 480 brain samples from people who died at ages ranging from 16 to 106 and who had not been diagnosed with neurodegenerative disease. (the-scientist.com)
Gene expression changed to a greater extent with age in glial cells than in neurons, the researchers found. (the-scientist.com)
Generally, neuron- and oligodendrocyte-specific genes showed decreased expression with age, while the expression of microglia-specific genes increased with age. (the-scientist.com)
The expression changes of astrocyte-specific genes was more complex-increasing with age in some brain areas and decreasing with age in others. (the-scientist.com)
To determine whether age-related changes in neuronal and glial cell numbers affected gene expression results, the researchers used imaging to count various cell types in tissue samples from the frontal cortices of young and older brains. (the-scientist.com)
Consistent with the gene-expression results, the number of oligodendrocytes decreased with age. (the-scientist.com)
The researchers concluded that the gene-expression changes in neurons and oligodendrocytes are attributable both to changes in cell number and to expression levels in individual cells. (the-scientist.com)
The expression of glial cell-specific genes was a better predictor of age than that of neurons, the researchers found, underscoring the relevance of non-neuronal cells to aging. (the-scientist.com)
As people aged, the gene expression patterns of glial cells in different brain regions became more and more similar, as if the glial cells were losing their region-based identities, the authors noted. (the-scientist.com)
Glial cells appear to have something of an identity crisis as we age, as their expression patterns become more similar across the brain," Ule and Patani wrote. (the-scientist.com)
Analyses of gene expression in cells affected by neurodegenerative disease can provide important insights into disease mechanisms and relevant stress response pathways. (frontiersin.org)
Isolated mDA neurons were subjected to genome-wide gene expression analysis by mRNA sequencing. (frontiersin.org)

Diagram shows steps involved in conducting gene therapy for sickle cell disease. (nih.gov)
Researchers at the National Institutes of Health have developed a new and improved viral vector-a virus-based vehicle that delivers therapeutic genes-for use in gene therapy for sickle cell disease. (nih.gov)
The development of the vector could make gene therapy for sickle cell disease much more effective and pave the way for wider use of it as a curative approach for the painful, life-threatening blood disorder. (nih.gov)
Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease," said study senior author John Tisdale, M.D., chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). (nih.gov)
This Request for Applications (RFA), Gene Therapy for Sickle Cell Disease, is related to the priority areas of clinical prevention services, chronic disabling conditions, and maternal and infant health. (nih.gov)
An important goal of this program is to attract new and established investigators into the field of gene therapy for sickle cell disease by providing access to critical technologies in the form of shared facilities and funds to pursue innovative pilot/feasibility studies. (nih.gov)
Britain's regulator said its decision to authorize the gene therapy for sickle cell disease was based on a study done on 29 patients, of whom 28 reported having no severe pain problems for at least one year after being treated. (wbay.com)
Dr Kanter, can you talk about the Mark Walters, MD, abstract and what we should look forward to with the gene therapy for sickle cell disease? (consultantlive.com)
A gene therapy for sickle cell anemia is in late clinical development and could be on the market soon. (medscape.com)

Somatic cell gene mutations in humans: biomarkers for genotoxicity. (nih.gov)
Specifically, we use a range of molecular, cellular and animal models to look at the effects of mutations on proteins and of variants or missing proteins on cells and on the function of the aging nervous system. (nih.gov)
And a subsequent study, presented in the July 14, 2005 issue of the New England Journal of Medicine from another laboratory that did not show correlations between gene mutations and patient survival (Volume 353:133-144 Number 2). (cancer.org)
When they put these cells into mice, those that were provoked to have higher levels of inflammation logged more mutations in their DNA. (newscientist.com)
By injecting the CRISPR protein into zebrafish embryos, they could use the random mutations they created to trace which cells go on to form which organs. (newscientist.com)
A team at Novartis has found that genome editing kills most human embryonic stem cells - and that the ones that do survive are likely to have mutations in a key anti-cancer gene. (newscientist.com)
The mutations that allow cancer cells to divide uncontrollably also make the cancer cells more dependent on certain cellular processes," notes senior author, Robert Weiss, associate professor of molecular genetics. (genengnews.com)
Researchers have linked newly discovered gene mutations to some cases of the progressive fatal neurological disease amyotrophic lateral sclerosis - ALS, also known as Lou Gehrig's disease. (nih.gov)
Shedding light on how ALS destroys the cells and leads to paralysis, the researchers found that mutations in this gene affect the structure and growth of nerve cells. (nih.gov)
Scientists at the University of Massachusetts Medical School, Worcester, collaborated with international ALS researchers to search for gene mutations in two large families with an inherited form of ALS. (nih.gov)
This deep sequencing of the exome led to the identification of several different mutations in the gene for profilin (PFN1) which were present only in the family members that developed ALS. (nih.gov)
Neural cells with PFN1 mutations also contained clumps of a protein known as TDP-43. (nih.gov)

Researchers have used virus-based vehicles for years in gene therapy experiments, where they have been very effective at delivering therapeutic genes to bone marrow stem cells in the lab before returning them to the body. (nih.gov)
For the past 30 years, researchers have been designing these beta-globin vectors in a reverse structural orientation, meaning the therapeutic genes incorporated into the virus are translated, or "read," from right to left by the viral vector-making machinery - much like reading an English sentence backwards. (nih.gov)
They found that the new vectors could transfer a much higher viral load - up to six times more therapeutic beta-globin genes than the conventional vectors - and had four to 10 times higher transduction efficiency, a measure of the ability to incorporate corrective genes into repopulating bone marrow cells. (nih.gov)
The multiplex assay will be used as a quality check on the cells, and for screening the effects of potentially therapeutic compounds. (nih.gov)
For example, therapeutic immune cells could be engineered to patrol a person's body, recording what they see and reporting back to clinicians when they are recaptured. (newscientist.com)
Therefore, it is key to identify the hub genes, explore the pathogenesis, and search for the therapeutic targets of AD. (hindawi.com)
The group's research could pave the way toward identifying potential therapeutic targets for stem cell-mediated regenerative medicine, as well as the treatment of bone disorders like osteoporosis, the most common type of metabolic bone disease. (news-medical.net)
British Library EThOS: Development of a cell-specific targeting strategy for therapeutic gene delivery vectors. (bl.uk)
Our study shows that, when we compare the costs of gene therapy and existing standard-of-care treatment for SCD using a technique that accounts for historical health disparities, gene therapy could be an equitable therapeutic strategy for all patients with SCD, whether their disease is mild, moderate, or severe," he said. (medscape.com)
She noted, however, that some patients start gene therapy for SCD at older ages and that it's important to analyze whether the treatment can still be cost-effective or the best therapeutic option for such patients. (medscape.com)

In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently used, and it had a carrying capacity of up to six times higher, the researchers report. (nih.gov)
The scientists have discovered two key epigenetic regulating genes that govern the cell-fate determination of human bone marrow stem cells. (news-medical.net)
Through our recent discoveries on the lineage decisions of human bone marrow stem cells, we may be more effective in utilizing these stem cells for regenerative medicine for bone diseases such as osteoporosis, as well as for bone reconstruction,' Wang said. (news-medical.net)
The new medicine, Casgevy, works by targeting the problematic gene in a patient's bone marrow stem cells so that the body can make properly functioning hemoglobin. (wbay.com)

Here, we show that NFAT targets MAPKs, histone acetyltransferase p300, and histone deacetylases (HDACs) to gene promoters to differentially regulate insulin and TNF-α genes. (nih.gov)
Moreover, inhibiting p38 and JNK induced a switch from NFAT-p38/JNK-histone acetyltransferase p300 to NFAT-ERK-HDAC3 complex formation upon the TNF-α promoter, which resulted in gene repression. (nih.gov)
He and his team found that KDM4B and KDM6B, two gene-activating enzymes, can promote stem cells' differentiation into bone cells by removing methyl markers from histone proteins. (news-medical.net)
To investigate this process we focused on the regulation of two late cell cycle genes, histone and thymidine kinase, in T2 cells and fibroblasts exposed in vitro to varying periods of hyperoxia (95% O2). (jci.org)

The stem cells Dr. Bharti is using to make RPE are induced pluripotent (iPS) stem cells, which are produced by reverting mature cells to an immature state, akin to embryonic stem cells. (nih.gov)
Embryonic stem cells could help treat all kinds of disorders, and editing the genomes of these stem cells could make the treatments far more potent. (newscientist.com)
Gene networks in human embryonic stem cells (hESCs) have been found to serve two purposes at once. (genengnews.com)
This discovery, from a study led by researchers from Brigham and Women's Hospital and Harvard Medical School (HMS), suggests that faulty embryonic stem cells have a built-in mechanism to ensure that they are destroyed before they can compromise the functioning of future cells and tissues. (genengnews.com)
Instead of looking at genes one by one, we looked at thousands of genetic alterations at the same time to determine how they affect the proliferation of embryonic stem cells, and, subsequently, the development of the three germ layers that serve as the raw material for human tissues. (genengnews.com)

Thus, the Freiburg researcher hopes to gain a better understanding of the extent to which noise regulates development or is even necessary for the differentiation of cells. (mpg.de)
Essential genes - such as transcription factors that mediate which genes should be switched on or off - are often only weakly expressed in cell differentiation, sometimes with high variability in cells of the same type. (mpg.de)
We assume that the fluctuating activity of these gene networks - in other words, the noise of these genes - could trigger the differentiation," says Dominic Grün. (mpg.de)
reported that hematopoietic stem and progenitor cells gave rise to iPSCs up to 300 times more efficiently than terminally differentiated B and T cells did, suggesting that the differentiation state of the starting cells affects reprogramming efficiency 23 . (nature.com)
The groundbreaking study grew out of Wang's desire to better understand the epigenetic regulation of stem cell differentiation, in which the structure of genes is modified while the sequence of the DNA is not. (news-medical.net)
The findings imply that chemical manipulation of these gene-activating enzymes may allow stem cells to differentiate specifically into bone cells, while inhibiting their differentiation into fat cells. (news-medical.net)
The research group, through its study of aging mice, found that the two enzymes KDM4B and KDM6B could specifically activate genes that promote stem cell differentiation toward bone, while blocking the route toward fat. (news-medical.net)
The researchers used genome-wide genetic screening to both overexpress and inactivate ("knock out") tens of thousands of genes that govern embryonic stem cell proliferation and differentiation into the three germ layers. (genengnews.com)

Results showed that mostly non-overlapping genes were differentially expressed in ILBD, subjects who were early after diagnosis (less than five years) and those autopsied at more advanced stages of disease (over five years since diagnosis). (frontiersin.org)
The identity of differentially expressed genes suggested that more resilient, stably surviving DA neurons were enriched in samples from advanced stages of disease, either as a consequence of positive selection of a less vulnerable long-term surviving mDA neuron subtype or due to up-regulation of neuroprotective gene products. (frontiersin.org)

Furthermore, maintaining preproinsulin mRNA levels depends not only on gene promoter activity, but also on heteronuclear mRNA processing and preproinsulin mRNA stability, the latter of which is likely ß-cell specific. (medscape.com)
Specific glucose-induced translational regulation of proinsulin synthesis appears to be unique to the ß-cell, with discrete signals interacting with cis elements in the untranslated regions of preproinsulin mRNA [ 28 ] . (medscape.com)
In addition, the mRNA vaccines developed by Pfizer and Moderna have led to improved cold-chain manufacturing, distribution, and storage capabilities throughout the sector, providing a much stronger foundation for cell and gene therapy products that have similar requirements. (bcg.com)
This involves measuring the number of mRNA molecules generated by active genes in the individual cells. (mpg.de)
Just like a kind of fingerprint, the number of different mRNA molecules per gene in a particular cell informs about the cell's identity and the relationship between the cells. (mpg.de)

SINGAPORE, Sept. 19, 2023 (GLOBE NEWSWIRE) -- Novotech , the leading Asia Pacific centred biotech CRO with global execution capabilities has just been awarded for the second consecutive year the Best CRO for Cell & Gene Therapy 2023 at the Asia Pacific Cell & Gene Therapy Excellence Awards . (woodtv.com)
FILE - This microscope photo provided on Oct. 25, 2023, by the Centers for Disease Control and Prevention shows crescent-shaped red blood cells from a sickle cell disease patient in 1972. (wbay.com)
Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. In a statement on Thursday, Nov. 16, 2023, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene editing tool CRISPR, which won its makers a Nobel prize in 2020. (wbay.com)
SINGAPORE , Sept. 18, 2023 /PRNewswire/ -- On September 14 , 2023, Biosyngen Pte Ltd (hereinafter referred to as "Biosyngen") was the recipient of the prestigious "Most Promising Cell Therapy Pipeline in APAC" award at the 7th Annual Cell & Gene Therapy World Asia 2023, amidst a pool of international biotechnology companies. (wowktv.com)
PALO ALTO, Calif. & RESEARCH TRIANGLE PARK, N.C.--( BUSINESS WIRE )-- Kriya Therapeutics, Inc. , a biopharmaceutical company developing gene therapies for conditions affecting millions of people around the world, today announced that it will present 10 abstracts at the 26th Annual Meeting of the American Society of Gene and Cell Therapy ( ASGCT ) taking place in Los Angeles, CA, May 16-20, 2023. (businesswire.com)

Sickle cell disease is an inherited blood disorder caused by a mutation, or misspelling, in the beta-globin gene (or β-globin gene). (nih.gov)
With gene therapy, doctors modify the patient's bone marrow hematopoietic (blood-producing) stem cells in the lab by adding a normal copy of the beta-globin gene through the use of a viral vector. (nih.gov)
In contrast to the old vector, the gene sequence, or "message," of the new beta-globin vector is read from left to right - like reading a normal sentence - making the gene translation approach less complicated, Tisdale explained. (nih.gov)
We also have a gene addition approach using a lentiviral vector, but instead of inserting the modified LentiGlobin beta-globin type, we're actually trying to use a different non-sickling globin. (consultantlive.com)

Despite this remarkable progress, it is clear that a significant number of genes that regulate circadian rhythms in mammals remain to be discovered and identified. (nih.gov)
Nuclear factor of activated T cells (NFAT) is activated by calcineurin in response to calcium signals derived by metabolic and inflammatory stress to regulate genes in pancreatic islets. (nih.gov)
Finally, the researchers observed that many of the genes that regulate the formation of the three germ layers also are known contributors to the growth of cancers when they are over- or underexpressed in somatic cells. (genengnews.com)

Apply through a new funding opportunity to use somatic cell gene editing (SCGE) approaches to improve graft survival and outcomes for recipients of allogenic solid organ, pancreatic islet, or vascularized composite allograft (VCA) transplants. (nih.gov)
NIAID and the National Heart, Lung, and Blood Institute (NHLBI) cosponsor the Notice of Special Interest (NOSI): Somatic Cell Gene Editing Therapies To Improve Transplantation Outcomes initiative. (nih.gov)
The reprogramming of somatic cells into induced pluripotent stem cells (iPSCs) by defined transcription factors has been a well-established technique and will provide an invaluable resource for regenerative medicine. (nature.com)

The GMP Gene Vector Lab produces clinical grade vectors for use in Phase I/II trials, while the GMP Clinical Research Lab prepares patient components for clinical trials. (wikipedia.org)
The initiative's goal is to advance safe, effective and durable gene-based cures to clinical trials in the U.S. and SSA within the next seven to 10 years. (nih.gov)
But even as the agents make impressive clinical progress-the alliance counts 1,220 clinical trials underway last year-cell and gene therapies continue to face big scientific, technological , and business-related hurdles. (bcg.com)
Looking to the future, he anticipates more successful clinical trials and the approval of more innovative cell and gene therapy which ultimately benefit a larger population of patients. (wowktv.com)
Enhancing good manufacturing practice (GMP), customer compliance and efficient aseptic manufacturing is essential as new advanced therapy medicinal products (ATMPs), including cell, gene and biological products advance through clinical trials to commercialisation. (pharmiweb.com)

Signals from the environment or from other cells activate proteins called transcription factors. (medlineplus.gov)
This finding is consistent with our current understanding that cancer usually involves defects in more than one critical gene, including defects that activate cancer-causing genes (oncogenes) like TCL1 and defects that inactivate cancer-preventing genes (tumor suppressors). (lymphomainfo.net)
Depending on type and stage of development, cells activate different genes, which are initially translated into RNA molecules that form the basis for the synthesis of proteins. (mpg.de)
In the new NCI-funded study, scientists found that brain cancer cells also slip several different genetic "on switches"-pieces of DNA that help activate genes-into these DNA circles. (cancer.gov)
These switches also activate EGFR in cells that lack amplification of the gene, they found. (cancer.gov)
Unlike the other two switches, these elements are typically isolated from EGFR on the chromosome and don't normally activate the gene. (cancer.gov)

By controlling the level of transcription, this process can determine when and how much protein product is made by a gene. (medlineplus.gov)
Scientists report they have created a tiny, nanosize container that can slip inside cells and deliver protein-based medicines and gene therapies of any size -- even hefty ones attached to the gene-editing tool called CRISPR. (nih.gov)
Integrating genetic variation with cell-type specific variation in transcript and protein abundance to make causal inferences about potential molecular targets. (nih.gov)
These include new alleles of known circadian genes as well as novel circadian loci such, Fbxl3, an orphan member of the F-box protein family, which we have found to interact selectively with the CRY proteins to target them for degradation through the proteasome pathway. (nih.gov)
Studies of humans who live longer than 100 years have shown that many share an unusual version of a gene called Forkhead box protein O3 (FOXO3). (sciencedaily.com)
This modification sends the protein into the nucleus of the stem cell where it turns on stress response genes. (sciencedaily.com)
This nutrient is needed to help a protein called lamin form a protective envelope around the DNA in the nucleus of the stem cell. (sciencedaily.com)
This gene encodes a multispan transmembrane protein belonging to the MAL proteolipid family. (nih.gov)
The protein is a component of lipid rafts and, in polarized cells, it primarily localizes to endosomal structures beneath the apical membrane. (nih.gov)
Dynamics of MAL2 during glycosylphosphatidylinositol-anchored protein transcytotic transport to the apical surface of hepatoma HepG2 cells. (nih.gov)
Overall, these data indicate that NFAT directs signaling enzymes to gene promoters in islets, which contribute to protein-DNA complex stability and promoter regulation. (nih.gov)
A gene is a section of DNA that contains information for making a protein. (cancer.gov)
Both sickle cell disease and thalassemia are caused by mistakes in the genes that carry hemoglobin, the protein in red blood cells that carry oxygen. (wbay.com)

Gene therapy trials using reverse-oriented vectors for sickle cell disease and beta-thalassemia have largely been encouraging, the researchers said, but this complicated gene translation process has made vector preparation and gene-transfer efficiency more difficult. (nih.gov)
Decades of basic research on sickle cell disease by scientists-including NIH-supported researchers- and selfless efforts by clinical trial participants have helped lay the groundwork for these novel genetic approaches. (nih.gov)
Researchers, patients, clinicians, and advocacy groups expect these new FDA-approved approaches to help people with sickle cell disease live longer, less painful, and more productive lives. (nih.gov)
We collaborated with researchers from Egypt, Syrian Arab Republic and Jordan in a study of patients with sickle-cell disease from those countries, and from various parts of Saudi Arabia, in order to investigate the influence of genetics on the clinical presentation of the disease, and to attempt to determine the origin of the sickle-cell gene in Arabs. (who.int)
Researchers at the National Institutes of Health have developed a technique that will speed up the production of stem-cell derived tissues. (nih.gov)
The technique will help the researchers in their efforts to use patients' skin cells to regenerate retinal pigment epithelium (RPE)-a tissue in the back of the eye that is affected in several blinding eye diseases. (nih.gov)
Once they had the assay working, the researchers halved its cost and increased its output by using fewer cells and smaller solution volumes. (nih.gov)
Researchers will report on the discovery of genetic defects that cause particular types of non-Hodgkin lymphoma (NHL), making these genes rational targets for new anti-lymphoma treatments, in the next issue of of the journal Blood. (lymphomainfo.net)
Researchers have found that defects in one gene (TCL1) can cause particular types of non-Hodgkin lymphoma. (lymphomainfo.net)
The researchers then engineered mice to aberrantly express the TCL1 gene and those mice developed B-NHLs at a very high rate, within the germinal centers of lymphoid tissues, such as lymph nodes and the spleen, where B cells normally develop. (lymphomainfo.net)
Using a mouse model, the researchers explored how cells respond when both genes are inhibited. (genengnews.com)
The researchers believe that the number and variety of switches are optimized to help the cancer cells survive and grow, Dr. Scacheri explained. (cancer.gov)

The genetic manipulation of the individual components is obtained by whole-body and conditional gene ablation in developing embryos and adult mice. (nih.gov)
In 2018, Dr. Paik and her team showed that mice who lack the FOXO3 gene in their brain are unable to cope with stressful conditions in the brain, which leads to the progressive death of brain cells. (sciencedaily.com)
By using the VarID method, the author was able to track the activity of essential transcription factors during the development of blood cells in mice. (mpg.de)
There are similar challenges when patient cells are transplanted into laboratory mice in order to conduct pre-clinical cancer research. (cedars-sinai.edu)
Less than 1% of patient cells grow and multiply in mice. (cedars-sinai.edu)
Intriguingly, the anti-tumor functions of METTL3 knockdown in the phenotype of NSCLC cells and xenograft mice were overturned by inhibition of SFRP2. (nature.com)

In the assay, tiny snippets of DNA tethered to beads are used to capture RNA molecules-created when genes are expressed by cells in the RPE sample. (nih.gov)
It serves as the basic instructions that allow you to create antibodies, or enzymatic proteins, or RNA molecules with your gene therapy. (wral.com)
To identify and exploit the cellular basis of health and disease we develop and apply single-cell multi-omics and spatial approaches, machine learning, lineage tracing, personalised organoid models and advanced imaging at scales ranging from molecules to individual cells to tissues and organisms. (mdc-berlin.de)

The concepts underlying cell death assays are relatively simple: if the drug kills tumor cells from an individual patient in a test tube, then the drug is more likely to be effective when administered to a patient. (cancer.org)
A vast diversity of individual tumor cells exists in every patient with cancer. (cedars-sinai.edu)
These leukemia models do not capture the full diversity of individual tumor cells within a single patient, let alone within the broader population of patients affected by this disease. (cedars-sinai.edu)
For example, in an aggressive type of brain cancer called glioblastoma , tumor cells typically have many copies of the EGFR oncogene . (cancer.gov)
To investigate the tumor cells' adaptation to hypoxia, we recreated two models using two lung cancer cell lines in the presence of intermittent hypoxia, which is characterized by changes in oxygen pressure within the disorganized vascular network. (nih.gov)

MAL2 promotes proliferation, migration, and invasion through regulating epithelial-mesenchymal transition in breast cancer cell lines. (nih.gov)
However, downregulation of METTL3 restrained the proliferation, migration and invasion of NSCLC cells. (nature.com)
Fig. 5: Suppression of SFRP2 abolished the potency of METTL3 knockdown in NSCLC cell proliferation, migration and invasion. (nature.com)
We investigated biological behaviors such as cell cycle, proliferation, radiation sensitivity, apoptosis and migration, hypoxia signal pathway in the lung cancer cells treated with chronic intermittent hypoxia, as well as the role of hypoxia inducible factor 1 there, hypoxia-inducible genes analyzed by real-time RT-PCR chip in H446 cells treated with the model. (nih.gov)
Inhibition of lung epithelial cell proliferation by hyperoxia. (jci.org)
Posttranscriptional regulation of proliferation-related genes. (jci.org)
The regulation of T2 cell proliferation and the effect of reactive oxygen (O2) species on this lung cell proliferation have not been well defined. (jci.org)
Hyperoxia for 24 to 48 h arrested cell proliferation in a SV40T-immortalized T2 cell line we have developed and in primary and SV40T-immortalized lung fibroblasts. (jci.org)
Thus proliferation arrest induced by hyperoxia was associated with posttranscriptional control of at least two late cell cycle-related genes. (jci.org)
This form of proliferation arrest is also seen when primary and SV40T-T2 cells but not fibroblasts are serum deprived, suggesting that T2 cells in vitro may be uniquely sensitive to alterations in their redox state and that these alterations in turn affect translational control of a subset of proliferation-related genes. (jci.org)

Novotech is also the recipient of the Best CRO for Cell & Gene Therapy 2022 award. (woodtv.com)
She remarked "according to our latest CGT clinical trial landscape report the global CGT market is projected to hit $93.78 billion by 2030 and FDA approvals for gene therapy in H2 2022 surpassed the combined total for the previous five years. (woodtv.com)
Notably, the FDA has granted approval to a greater number of gene therapy drugs in the latter half of 2022 alone compared to the prior five-year period. (wowktv.com)

That treatment, the company's flagship gene therapy in neuroscience, Zolgensma, was approved in May 2019 in the U.S. to treat children less than two years old with SMA. (wral.com)

THIS March, gene therapy made a significant breakthrough-scientists at the Oregon Health & Science University in Portland, United States, used the gene-editing tool Crispr-Cas9 to edit DNA inside a human body for the first time, also called in vivo gene editing. (deloitte.com)
We'll soon know - the CRISPR gene editing technique has been adapted to make cells keep a log of what happens to them, written inside their own DNA. (newscientist.com)
Such CRISPR-based logging could have a huge range of uses, from smart cells that monitor our health from within, to helping us understand exactly how our bodies develop and grow. (newscientist.com)
They designed a system where CRISPR can be activated within a cell whenever it experiences a particular event - for example, exposure to a particular chemical. (newscientist.com)
CRISPR-based logging could help us understand what happens inside cells that turn cancerous, for instance, or help us monitor the levels of substances in the body. (newscientist.com)
And North Carolina State University's Rodolphe Barrangou, Ph.D., uses a different CRISPR enzyme to edit genes. (wral.com)
In a statement Thursday, the Medicines and Healthcare Regulatory Agency said it approved Casgevy, the first medicine licensed using the gene-editing tool CRISPR, which won its makers a Nobel prize in 2020 . (wbay.com)
The future of life-changing cures resides in CRISPR based (gene-editing) technology," said Dr. Helen O'Neill of University College London. (wbay.com)

However, in the long term such translational control is supplemented by regulation at the transcriptional level and by insulin degradation within the ß-cell. (medscape.com)
In mammals, circadian oscillations are generated by a set of genes forming a transcriptional autoregulatory feedback loop: these include: Clock, Bmal1, Per1, Per2, Cry1, Cry2 and Casein kinase 1 epsilon. (nih.gov)
Furthermore, the data suggest that TNF-α can be repressed and insulin production can be enhanced by selectively targeting signaling components of NFAT-MAPK transcriptional/signaling complex formation in pancreatic β-cells. (nih.gov)

Tracing cell lineages is crucial for understanding how our genomes develop bodies from a single cell. (newscientist.com)
There are other methods for tracing cell lineages, but these all have limitations, and can involve spending many years painstakingly watching embryos under a microscope . (newscientist.com)
This process occurs through the activation of certain genes favoring a commitment to one lineage and the concurrent deactivation of genes favoring other lineages. (news-medical.net)

iPS cells can be derived from a patient's skin or blood cells, coaxed into other cell types (such as neurons or muscle), and in theory, re-implanted without causing immune rejection. (nih.gov)
This causes the stem cell to go dormant and stop producing new neurons. (sciencedaily.com)
This response is actually very good for the stem cells because the outside environment is not ideal for newly born neurons," Dr. Paik explained. (sciencedaily.com)
It's better for stem cells to remain dormant and wait until the stress is gone to produce neurons. (sciencedaily.com)
ALS attacks motor neurons, the nerve cells responsible for controlling muscles. (nih.gov)
In fly models, disrupting profilin stunts the growth of axons - the long cell projections used to relay signals from one neuron to the next or from motor neurons to muscle cells. (nih.gov)
Motor neurons are large cells with long axons that connect to muscle, and cytoskeleton proteins are especially important in the transport of proteins along the axon to the remote parts of the neuron. (nih.gov)
Typically we have concentrated on neurons for studies of dementia, as they are the cells involved in brain processing and memories. (the-scientist.com)
Based on previous data about the genes expressed by different cell types, the team sorted the expressed genes into those specific to neurons and those specific to glia. (the-scientist.com)
The spinal cord neurons are shown in a blue tone, the skeletal muscle cells are marked purple. (mdc-berlin.de)

The composition of preproinsulin cDNA to be used for gene therapy vectors thus requires careful attention, but may not itself be sufficient to allow for translational control in a surrogate ß-cell. (medscape.com)

At the end of the culture period, a measurement is made of cell injury, which correlates directly with cell death (apoptosis). (cancer.org)
If a cell has an error in its DNA that cannot be repaired, it may undergo self-destruction ( apoptosis ). (medlineplus.gov)
Apoptosis is a common process throughout life that helps the body get rid of cells that no longer work or that it doesn't need. (medlineplus.gov)
Apoptosis protects the body by removing genetically damaged cells that could lead to cancer, and it plays an important role in the development of the embryo and the maintenance of adult tissues. (medlineplus.gov)
Paraquat-induced apoptosis in human neuroblastoma SH-SY5Y cells: involvement of p53 and mitochondria. (nih.gov)
They maintain pluripotency, and they keep apoptosis, or programmed cell death, on a hair trigger. (genengnews.com)
When genes known to control pluripotency were deleted-genes such as OCT4 and SOX2-the hESCs surprisingly increased their resistance to death, indicating that under normal circumstances pluripotency regulators also contribute to apoptosis pathways. (genengnews.com)
The cells mediated by irradiation induced resistance to radiation and apoptosis and increased metastasis in lung cancer cells. (nih.gov)

Progress in stem cell-based therapies has been limited by our capacity to authenticate cells and tissues," said Kapil Bharti, Ph.D., a Stadtman Investigator in the Unit on Ocular and Stem Cell Translational Research at the National Eye Institute (NEI), a part of NIH. (nih.gov)
Using circadian gene reporter methods, one can demonstrate that most peripheral organs and tissues can express circadian oscillations in isolation, yet still receive and may require input from the dominant circadian pacemaker in the suprachiasmatic nucleus (SCN) in vivo. (nih.gov)
When they inactivated the Hus1 gene in healthy mammary gland tissues, it caused genome damage and cell death. (genengnews.com)
The manufacturing step differs depending on whether the prospective therapy is autologous, or based on cells or tissues from a patient, or allogeneic where cells are taken from different individuals of the same species and prepared for the patient. (onenucleus.com)

Gene Ontology (GO) and Kyoto Encyclopedia of Genes and Genomes (KEGG) pathway enrichment analyses were performed on the 119 DEGs, revealing some important biological functions and key pathways. (hindawi.com)

The ability to control whether certain stem cells ultimately become bone cells holds great promise for regenerative medicine and potential therapies aimed at treating metabolic bone diseases. (news-medical.net)
As pluripotent cells-progenitors of every type of cell type in the body-hESCs are of central interest to developmental and regenerative biologists. (genengnews.com)

They also found that mutant profilin accumulated in clumps in neural cells, as has been seen for other abnormal proteins associated with ALS, Parkinson's and Alzheimer's. (nih.gov)
For example, BlueRock Therapeutics LP is using induced pluripotent stem cells (iPSCs) as source material for a stem cell therapy for Parkinson's disease. (onenucleus.com)

The National Human Genome Research Institute provides a definition of gene regulation in their Talking Glossary of Genetic Terms. (medlineplus.gov)
The sickle cell patients are subjected to several factors causing inflammation, and the genetic identification of each individual allows to focus the possibility of allelic variations influence of a specific gene and then the polymorphism. (nih.gov)
Globally, cell and gene therapies (CGT) are transforming not just how humans treat genetic and intractable diseases but are altering the entire pharmaceutical ecosystem. (deloitte.com)
In recent years, gene-based treatments have been groundbreaking for rare genetic disorders and infectious diseases," said Dr. Trevor Mundel, president of global health at the Gates Foundation. (nih.gov)
Gene therapy alleviates the underlying cause of genetic diseases and acquired diseases. (wral.com)
Rather than treating symptoms, gene therapy works by repairing or enhancing cells at the genetic level. (wral.com)
At Novartis Gene Therapies, the company's goal is to develop and commercialize gene therapies for patients and families affected by rare and life-threatening neurological genetic diseases. (wral.com)
Lentiviral vector is the predominant delivery vehicle used for introducing genetic modifications into cell therapies such as CAR-T and HSC gene therapies. (lonza.com)
When the cell cycle proceeds without control, cells can divide without order and accumulate genetic errors that can lead to a cancerous tumor . (medlineplus.gov)
Twenty-three gene therapies have been FDA-approved with promising results for treating diseases including blood cancers, hemophilia, congenital blindness, and spinal muscular atrophy, which is the leading genetic cause of death in infants. (mckinsey.com)
While the exact cause of AD has not been elucidated, studies have found that AD is the result of a combination of genes, lifestyle, and environmental factors, caused in part by specific genetic changes [ 6 - 9 ]. (hindawi.com)
In people with sickle cell - which is particularly common in people with African or Caribbean backgrounds - a genetic mutation causes the cells to become crescent-shaped, which can block blood flow and cause excruciating pain, organ damage, stroke and other problems. (wbay.com)
Patients first receive a course of chemotherapy, before doctors take stem cells from the patient's bone marrow and use genetic editing techniques in a laboratory to fix the gene. (wbay.com)
Last year, Britain approved a gene therapy for a fatal genetic disorder that had a list price of £2.8 million ($3.5 million). (wbay.com)

Our analysis shows that, in the decade leading up to 2020, six major pharma companies alone made more than 50 cell and gene therapy alliances and licensing deals worth over $16 billion and spent approximately $38 billion on acquisitions. (bcg.com)
In 2020, six cell and gene therapy programs were forced to delay their development timelines after the FDA asked for more information about their production processes. (bcg.com)

We develop general operating principles and conduct gene network modeling based on genome-wide experimental data. (nih.gov)

Advances in analysis of single-cell technologies, while still in development, are leading investigators to map the cellular diversity of the brain at single-cell resolution (e.g. (nih.gov)
It's a new wave of treatments that we can utilize for patients with sickle cell disease," said Dr. James LaBelle, director of the pediatric stem cell and cellular therapy program at the University of Chicago. (wbay.com)
The biggest challenge we face is the incomplete understanding of the cellular mechanisms that drive cells through normal development and the pathological and environmental changes that cause them to deviate towards disease. (mdc-berlin.de)

Cell and gene therapies have proved their potential only in rare diseases with well-defined genomic targets, high unmet need, and small numbers of patients. (bcg.com)
These genes may provide new targets for future therapies. (cedars-sinai.edu)

The U.S. Food and Drug Administration approved two new gene therapies to treat sickle cell disease - one a gene-editing and the other a gene-addition approach. (nih.gov)
Based in California with a location currently under construction in Sanford, Audentes develops gene therapies to treat conditions like X-Linked myotubular myopathy, Pompe disease, Duchenne muscular dystrophy and myotonic dystrophy. (wral.com)

At Audentes, the company has a similar focus, utilizing gene therapies to target life-threatening and often rare diseases. (wral.com)

These proteins bind to regulatory regions of a gene and increase or decrease the level of transcription. (medlineplus.gov)

There are few disease-modifying and life-extending therapies for those living with sickle cell disease, leaving patients burdened by chronic pain, high financial costs, health complications, and higher risk of early mortality. (nih.gov)
Dr. Bharti and colleagues at NEI are collaborating with the New York Stem Cell Foundation to produce iPS cell lines from patients with age-related macular degeneration (AMD)-the most common cause of vision loss among older Americans-and from other patients with rare hereditary eye diseases. (nih.gov)
One of the most exciting areas of innovation is Cell and Gene Therapy, with many therapies already changing the industry and patients' lives. (accenture.com)
HCPs can log in to Unify to onboard their Cell & Gene patients to sponsor therapies for which they are qualified. (accenture.com)
By comparison, the allogeneic approach relies on a single source of cells to treat many patients and is often described as an 'off-the-shelf' approach. (onenucleus.com)
Every consultant that would potentially be involved with that patient's care was trained and so when we were calling, we actually had a dedicated CAR-T line, a cell phone for CAR-T patients, and so that communication went directly from the principal investigator (PI) to the attending physician and the team," she said. (centerwatch.com)
The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. (wbay.com)
Patients must be hospitalized at least twice - once for the collection of the stem cells and then to receive the altered cells. (wbay.com)
In the study for thalassemia, 39 out of 42 patients who got the therapy did not need a red blood cell transfusion for at least a year afterward. (wbay.com)
All the way through from A to C, we know that it's persistent, so some of our patients who were in that first group are now 3, 3-and-a-half years out and they have that same amount of gene, of that new gene being expressed. (consultantlive.com)
The other big excitement about gene therapy is that it doesn't have the problems that we spoke about earlier in terms of stem cell transplant, and that all patients don't need a donor, they're their own donor. (consultantlive.com)
As a global Biotech headquartered in Singapore and China , Biosyngen is steadfast in its dedication to the development of innovative and impactful cell and gene therapy products, with the aim of benefiting patients worldwide. (wowktv.com)
However, it has the potential to be a cost-effective therapy for patients with sickle cell disease (SCD) in the United States, say investigators who analyzed the costs using a novel method that takes into account historical heath inequities. (medscape.com)
Goshua and colleagues included equity weight in an analysis of 10 years of data on annual healthcare costs for patients with SCD who were covered by private insurance and were treated with medications (for example, hydroxyurea), antibiotics, blood transfusion, and hematopoietic stem cell transplants. (medscape.com)
The estimate was based on the cost of US Food and Drug Administration-approved gene therapies, and it was assumed that the therapy would result in permanent disease remission for all patients. (medscape.com)
In addition, the model assumed that all eligible patients in the US with SCD who are aged 12 years and older would be offered the gene therapy. (medscape.com)

Diet plays a critical role in human health and disease and understanding the underlying molecular mechanisms by which dietary components influence cell biology and physiology is essential. (nih.gov)
3 Department of Developmental and Cell Biology, University of California, Irvine, CA 92617, United States of America. (nih.gov)
Many scientists thought that simply having several copies of an oncogene could cause a cell to become cancerous, explained Ian Fingerman, Ph.D., of the DNA and Chromosome Aberrations Branch in NCI's Division of Cancer Biology , who wasn't involved in the study. (cancer.gov)

Over a dozen genes have been linked to ALS, and these findings support existing studies which suggest that cell cytoskeleton disruptions play a major role in ALS and other motor neuron diseases. (nih.gov)

Though sickle cell disease (SCD), a genetically inherited disease, and HIV, an acquired infectious disease, present significantly different scientific challenges, gene-based treatments hold promise for both. (nih.gov)
By working with the NIH and scientists across Africa, we aim to ensure these approaches will improve the lives of those most in need and bring the incredible promise of gene-based treatments to the world of public health. (nih.gov)
While these treatments are critical to people with serious conditions, the potential of cell and gene therapies must be expanded to apply to diseases in larger indications (such as neurological disorders, diabetes, and cardiovascular illness) in order to maintain funding, resources, and attention. (bcg.com)
Using gene and cell therapies, companies like Locus Biosciences, Novartis Gene Therapies and Audentes, an Astellas company, are working to create cures and treatments for life-threatening conditions. (wral.com)
Gene and cell therapies are paving the way to breakthrough treatments and potential cures for serious illnesses and diseases, from spinal muscular atrophy to Pompe Disease. (wral.com)
Cell and gene therapies are next-generation treatments for a number of diseases. (mckinsey.com)
Gene therapy treatments can cost millions of dollars and experts have previously raised concerns that they could remain out of reach for the people who would benefit most. (wbay.com)

Compared to cultured RPE, iPS-derived RPE expressed higher levels of SOX2 and PAX6-both developmental markers-but lower levels of the RPE-specific genes, suggesting a relatively immature state. (nih.gov)
Scientists call this phenomenon "biological noise" and suspect that it is also decisive for the fate of cells, i.e. the developmental pathway a cell takes. (mpg.de)
The measurements can be used to analyze the spatial arrangement of individual cells in the tissue and to identify developmental trajectories and transitional states on these trajectories. (mpg.de)

Comparative non-cholinergic neurotoxic effects of paraoxon and diisopropyl fluorophosphate (DFP) on human neuroblastoma and astrocytoma cell lines. (nih.gov)

With this foundation, North Carolina's strengths in biological manufacturing naturally created a cluster of gene and cell therapy manufacturing, including Novartis Gene Therapies, Audentes, an Astellas company, and more. (wral.com)
Previous studies have shown that cancer cells could behave in the form of adaptation to hypoxia in tumor growth, which could result in their biological changes and determine their responses to the therapies. (nih.gov)

That discovery led Dr. Jihye Paik, associate professor of pathology and laboratory medicine at Weill Cornell Medicine, and her colleagues to investigate how this gene contributes to brain health during aging. (sciencedaily.com)
Our methods allowed us to create an 'atlas' of nearly every gene in the human genome and determine what its overexpression or loss does to the most fundamental first steps of human development," said Kamila Naxerova, PhD, a former postdoctoral fellow in the Elledge laboratory and the lead author of the Genes and Development article. (genengnews.com)

In silico methods could be utilized to leverage the strengths of high-depth multi-omic information generated from bulk tissue brain samples in conjunction with the more precise genomic information provided by single-cell approaches. (nih.gov)

In all of the causative genes that we identify, we look for common pathways," Dr. Landers said. (nih.gov)

The goal of this initiative is to encourage research that will advance our understanding of brain cell type-specific gene regulation to elucidate the pathogenesis of mental disorders. (nih.gov)

Their new study, published Jan. 28 in Nature Communications , reveals that FOXO3 preserves the brain's ability to regenerate by preventing stem cells from dividing until the environment will support the new cells' survival. (sciencedaily.com)
Cancer cells go to great lengths to sustain their growth, survival, and spread. (cancer.gov)

The findings, published November 27 in Cell , support a different way of thinking about cancer, said Peter Scacheri, Ph.D., of Case Western Reserve University. (cancer.gov)

Once captured, the RNA from distinct genes is labeled with a fluorescent tag. (nih.gov)
Those cells display distinct behaviors related to cancer growth, metastasis and responses to chemotherapy. (cedars-sinai.edu)

The Center for Cell and Gene Therapy conducts research into numerous diseases, including but not limited to pediatric cancers, diabetes, HIV, glioma and cardiovascular disease. (wikipedia.org)
For example, AlloVir, a Boston-based company developing cell therapies targeting life-threatening viral diseases, recently announced it is developing allogeneic T-cell therapies for COVID-19 in collaboration with the Baylor College of Medicine. (deloitte.com)
NIH and the Bill and Melinda Gates Foundation have announced they will invest $100 million each to develop affordable gene-based cures for the two diseases and make them globally available, including in low-resource settings. (nih.gov)
Many diseases, such as cancer, arise because cells do not fully develop from the stem cell to maturity. (mpg.de)
It aims to treat diseases by replacing, inactivating or introducing genes into cells- either inside the body, or in vivo, or outside of the body, or ex vivo,' said Jimmy Weirich, vice president and site head at Novartis Gene Therapies, a global healthcare company with a major manufacturing facility in Durham. (wral.com)
Disruption of normal regulation of the cell cycle can lead to diseases such as cancer. (medlineplus.gov)
In lay terms, cell and gene therapies restore, alter, or replace cells or genes in the body to prevent, treat, and sometimes cure diseases-some of which were previously incurable. (mckinsey.com)
Genes, Cells and Cell-based Medicine uses a combination of innovative experimental and computational approaches to understand the function and dysregulation of the genome and molecular networks in individual cells - the origin of most diseases. (mdc-berlin.de)
Kriya's abstracts include two oral presentations and eight posters on its core technology and manufacturing platforms as well as its diverse pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases. (businesswire.com)
Kriya Therapeutics, Inc. is a biopharmaceutical company developing gene therapies for diseases affecting millions of people around the world. (businesswire.com)
With operations in Palo Alto, California and Research Triangle Park, North Carolina, Kriya has raised over $450 million to advance a broad pipeline of gene therapies for ophthalmology, neurology, and metabolic diseases. (businesswire.com)

Our goal in this series, as well as the e-book on which they are based, has been to provide insights on current logistics best practices for global cell and gene therapy studies. (worldcourier.com)
Moreover, the technology also allows further insights into the cell. (mpg.de)

It is thus a prime opportunity to integrate new single-cell datasets with existing bulk tissue brain data resources such as GTEx, ENCODE, and the PsychENCODE Consortium (PEC). (nih.gov)

Retinal pigment epithelium derived from induced pluripotent stem cells. (nih.gov)

We have made some exciting research advances over the years and are ready to collect on our scientific investments in sickle cell research," Gibbons said. (nih.gov)

The Center for Cell and Gene Therapy is a translational research institute within Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, all of which are located in the Texas Medical Center in Houston, Texas. (wikipedia.org)

Anatomy29

Organisms12

Diseases5

Chemicals and Drugs32

Analytical, Diagnostic and Therapeutic Techniques and Equipment26

Phenomena and Processes45

Disciplines and Occupations1

Information Science3

Health Care2

Expression75

Therapy for sickle9

Mutations12

Therapeutic10

Bone marrow stem cells4

Histone4

Embryonic stem5

Differentiation8

Differentially2

MRNA5

20235

Beta-globin4

Regulate3

Somatic3

Clinical trials5

Activate6

Protein13

Researchers12

Mice6

Molecules3

Tumor cells5

Proliferation10

20223

20191

CRISPR8

Transcriptional3

Lineages3

Neurons10

Vectors1

Apoptosis8

Tissues4

Ontology1

Regenerative2

Parkinson's2

Genetic14

20202

Genome-wide1

Cellular3

Targets2

Therapies to treat2

Utilizing gene therapies1

Proteins1

Patients16

Biology3

Neuron1

Treatments7

Developmental3

Neuroblastoma1

Biological2

Laboratory2

Genomic1

Pathways1

Pathogenesis1

Survival2

Findings1

Distinct2

Diseases11

Insights2

Datasets1

Pluripotent1

Sickle cell research1

Translational research1