Suggests that gene therapy. Medical search. Frequent questions

The industry-funded clinic trial with Ultragenyx is evaluating UX701, an investigational adeno-associated virus (AAV) vector-based gene therapy. (ucdavis.edu)
Here, based on significant liver contribution to the catabolism of the branched chain amino acid leucine, we develop a liver-directed adeno-associated virus (AAV8) gene therapy for MSUD. (nature.com)
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)
For gene delivery, adeno-associated virus (AAV)-vectors are widely used gene delivery vectors for gene therapy due to features such as tissue tropism, potential of gene transfer to non-dividing cells, and long-term expression. (sbir.gov)
A DMD gene therapy based on an adeno-associated virus (AAV) delivery platform, Sarepta Therapeutics' SRP-9001, is currently under review at the U.S. Food and Drug Administration (FDA) and rival candidates are in clinical development. (scienceboard.net)
PerkinElmer has announced the launch of its 'ready-to-use' adeno-associated virus vectors detection kits to support researchers working on gene therapies. (scienceboard.net)

Working with scientists at the University of Missouri, Columbia, Davidson's team focused on the late infantile form of the disease that starts in children 2 to 4 years of age and is most often caused by mutations in the gene for the soluble lysosomal enzyme tripeptidyl peptidase 1 (TPP1), an enzyme which degrades proteins. (nih.gov)
Gene therapy has been utilized successfully to repair or inactivate mutations in animal models of monogenic human diseases 19 as well as human in vitro cells 12 . (nature.com)
Mutations in the otoferlin gene are linked to severe congenital hearing loss, a common type of deafness in which patients can hear almost nothing. (reachmd.com)
After that, we took a step back, and the new question became: When you lose this gene, when you have mutations, is expression of other genes affected? (reachmd.com)
Findings from experiments conducted so far suggest that the specific defects could be small inframe deletions, inframe additions, or point mutations. (medscape.com)
The differences in the clinical courses of types A and B suggest that the mutations are different. (medscape.com)
Tyrosine kinase inhibitors (TKIs) targeting epidermal growth factor receptor (EGFR), including gefitinib and erlotinib, have become the standard first-line therapy for patients with advanced non-small cell lung cancers (NSCLCs) harboring activating EGFR mutations ( 4 , 5 ). (spandidos-publications.com)
Of those in between, patients with low TMB and high interferon gamma signature did particularly well with dabrafenib/trametinib therapy versus placebo, which really makes you wonder whether patients with high TMB simply had other mutations that could potentially harm the impact of dabrafenib/trametinib adjuvant therapy. (medscape.com)
This gives more support to the idea that you can combine these different gene expression profiles and mutations. (medscape.com)
Since the identification of somatic mutations in the BRAF oncogene, targeted therapies directed toward mutated tumors have emerged and demonstrated dramatic clinical effect. (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)
For years, researchers have known that a gene called EAK-7 plays an important role in determining how long worms will live. (innovitaresearch.com)
The researchers began studying EAK-7, the worm gene, in 2013, when Krebsbach was a faculty member at the University of Michigan. (innovitaresearch.com)
The gene actually activated another biological process, which the researchers call an alternative pathway, one that was not as well understood at the molecular level as mTOR. (innovitaresearch.com)
As part of the trial, researchers used CRISPR-Cas9 to edit specific genes in stem cells-the building blocks of blood cells-taken from each patient. (medicalxpress.com)
However, researchers at UC Davis Health have infused their first Wilson Disease patient with a gene therapy as part of a groundbreaking new clinical trial. (ucdavis.edu)
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)
Meanwhile, researchers announced more miraculous cures of patients with rare and life-threatening diseases who were treated with experimental therapies. (technologyreview.com)
In March, researchers announced that a teenage boy in France had been cured of sickle-cell disease after receiving an experimental gene therapy developed by Bluebird Bio. (technologyreview.com)
In collaboration with Julianna Lisziewicz, Ph.D., of the Research Institute for Genetic and Human Therapy in Washington, D.C., the researchers took advantage of the fact that an HIV gene called tat is essential for the virus to replicate in the infected cells. (sciencedaily.com)
Using genetic engineering techniques, the researchers inserted the antitat gene into a mouse retrovirus that can enter cells that are potential sites for HIV replication. (sciencedaily.com)
After identifying the problem, researchers introduced the element into a lentiviral vector, successfully and significantly improving the quality and quantity of the gene therapy. (health.am)
image: Researchers developed nanoparticles that carry a gene editing package specifically to the liver in mice, successfully reducing levels of LDL 'bad' cholesterol in the blood. (eurekalert.org)
The Tufts and Broad researchers, however, have modified one gene that could provide a protective effect against elevated cholesterol if it can be shut down by gene editing. (eurekalert.org)
The gene that the researchers focused on codes for the angiopoietin-like 3 enzyme (Angptl3). (eurekalert.org)
If researchers can knock out the Angptl3 gene, they can let the lipases do their work and reduce levels of cholesterol in the blood. (eurekalert.org)
California: In a breakthrough study, researchers have found a potential treatment for life-threatening cardiac diseases by using gene therapy. (siasat.com)
Since the first successful gene therapy for thalassemia major, in 2007, researchers have worked to improve the efficacy and safety of the procedure. (medscape.com)
Researchers have conducted a study using gene therapy as a potential treatment for alcohol use disorder (AUD), revealing that sustained release of hGDNF in the brain might prevent relapse into excessive drinking. (yalibnan.com)
April 28, 2023 -- Researchers have created a lentiviral gene therapy vector capable of targeting muscle cells to treat the rare disease Duchenne muscular dystrophy (DMD) in mice. (scienceboard.net)

The findings from a new clinical trial, published August 31, add to the body of evidence supporting gene therapy as a treatment for sickle cell disease, which primarily impacts people of color. (medicalxpress.com)
The University of Chicago Medicine Comer Children's Hospital was one of three sites to enroll patients in the clinical trial, which tested a stem cell gene therapy to treat sickle cell disease. (medicalxpress.com)
Here we show efficacy of gene therapy for MSUD demonstrating its potential for clinical translation. (nature.com)
As an autosomal recessive monogenic disease, MSUD represents an ideal target for liver-directed gene therapy since clinical OLT data suggests that incomplete restoration of liver BCKD enzyme activity (representing 9-13% of body BCKD activity 10 ) is fully therapeutic. (nature.com)
In December, the company published early clinical trial results showing that nine patients who received its therapy saw substantial increases in the blood-clotting proteins absent in hemophilia. (technologyreview.com)
To investigate the in vitro effects of suicide gene therapy system of herpes simplex virus thymidine kinase gene (HSV-TK) in combination with the treatment of nucleotide analog-ganciclovir (GCV) on human pancreatic cancer, and to provide a novel clinical therapeutic method for human pancreatic cancer. (wjgnet.com)
As more cell and gene therapies enter into the clinical and commercial space, there is an emerging need for more cost-effective and scalable viral vector manufacturing platforms, such as lentiviral vector production. (thermofisher.com)
Last year marked the birth of gene therapy 2.0, in which the experimental dream finally became a clinical reality. (singularityhub.com)
It turns out that some lucky people have a natural mutation in their Angptl3 gene, leading to consistently low levels of triglycerides and low-density lipoprotein (LDL) cholesterol, commonly called "bad" cholesterol, in their bloodstream without any known clinical downsides. (eurekalert.org)
The gene therapy involves repairing of defective gene in vivo or delivering the gene product to target cells by clinical safe vehicle. (medgadget.com)
The introduction of gene therapy has shown potential to offer patients with eligible rare genetic diseases a transformational clinical benefit and improve quality of life. (medgadget.com)
Currently, there are more than 2000 cell and gene therapy clinical trials ongoing globally which are evaluating the role of about 500 gene and cell therapy products and have shown promising response. (medgadget.com)
Amid region, US is expected to witness significant growth in gene therapy market which is due to clinical trials combined with presence of key companies in the region. (medgadget.com)
Meanwhile, the acquisition of Prevail Therapeutics will add pre-clinical and clinical-stage gene therapy programs to Lilly's pipeline, a new and attractive avenue. (zacks.com)
Our expanding gene therapy portfolio is designed for a smooth ramp-up to clinical production and we have made focused investments to ensure we stay ahead of commercial demand. (pharmiweb.com)
Therefore, it is biologically reasonable that CYP19A1 rs1008805 (A/G) polymorphism may be associated with the clinical outcome of hormone therapy. (spandidos-publications.com)
In conclusion, the homozygous minor allele (GG) of CYP19A1 rs1008805 was identified to be significantly associated with an inferior clinical outcome of hormone therapy in postmenopausal hormone receptor‑positive patients with early breast cancer. (spandidos-publications.com)
The presence and intensity of ER and/or PgR are useful predictive markers for the response to hormone therapy in clinical practice. (spandidos-publications.com)
Pharmacogenomics can also be used in a broader sense for drug discovery and development, with increasing evidence suggesting that genomically defined targets have an increased success rate during clinical development. (cdc.gov)
Eight other healthy infants were diagnosed with gene variants with moderate probability of clinical disease and variable expressivity for other conditions, mostly various forms of cardiomyopathy, which might manifest in childhood. (cdc.gov)

the agency is expected to make a decision early next month, before considering another sickle cell gene therapy. (whnt.com)

Lentiviral (LV) vectors of the Retroviridae family show interesting properties for monogenic gene therapy, since they integrate into the host genome and allow long-lasting gene expression 22 . (nature.com)
One approach uses modified lentiviruses as vectors to deliver therapeutic genes to the lungs. (imperial.ac.uk)
But adenovirus vectors don't last long, so therapy must be frequently re-administered. (health.am)
Ultimately, introducing GRPE elements into viral vectors could enhance the ease and effectiveness of gene therapy, which typically uses transplanted human stem cells. (health.am)
AAV-vectors are currently known as the most advanced gene delivery vector and are used to transduce therapeutic genes to the CNS site for treatment of neurodegenerative disorders. (sbir.gov)
Dr. Ho did her PhD work in herpesvirus pathogenesis and postdoctoral research in CNS gene therapy with viral vectors at Stanford University. (stanford.edu)
This ®nding will lead to the development of lentiviral vectors with autoregulatory capacities that may be very useful for gene therapy to the brain. (lu.se)
The use of viral vectors to deliver foreign genes to the brain is highly logical states in the human brain (Eng et al. (lu.se)
2003). The therapeutic effects of different genes delivered by ment of the hGFAP promoter used here has been extensively studied and lentiviral vectors have been documented in a number of animal found to be up-regulated after several different stimuli both in vitro and models, both in rodents and primates, and examples include delivery in vivo in transgenic mice (Brenner et al. (lu.se)

As the world leader in serving science, Thermo Fisher Scientific delivers innovative solutions to accelerate the development of lentivirus production to help advance the field of cell and gene therapy. (thermofisher.com)
For gene therapy developers seeking to rapidly and efficiently scale production, a set of solutions is now available from Thermo Fisher Scientific to support adeno-associated viral (AAV) manufacturing. (pharmiweb.com)
Despite the immense promise of gene therapy, robustness and yield continue to pose challenges in the manufacturing process," said Betty Woo, vice president and general manager, cell and gene therapy at Thermo Fisher Scientific. (pharmiweb.com)

Gene therapy is an experimental form of treatment that uses genes in order to cure diseases and disorders by inserting genes into cells of the infected individual. (punnettssquare.com)
This is a huge break though for gene therapy, as it is expanding the different diseases and disorders that are able to be cured with this treatment. (punnettssquare.com)
It is also a breakthrough for scientists researching for other diseases, since this gene therapy can become a possible form of treatment. (punnettssquare.com)
I personally find for this to be very exciting, as now we can cure diseases and disorders a lot more efficiently with more research into gene therapy. (punnettssquare.com)
Findings from the study could be a preliminary step toward new therapies that would work by slowing or blocking mEAK-7's molecular process, which in turn could potentially control the spread and growth of the cells responsible for those diseases. (innovitaresearch.com)
At UChicago Medicine, we've built infrastructure to support new approaches to sickle cell disease treatment and to bring additional gene therapies for other diseases. (medicalxpress.com)
As the scientific community continues to refine and expand the applications of gene therapy, the potential for curative treatments for diseases like sickle cell disease is becoming more of a transformative reality. (medicalxpress.com)
The data from this trial supports bringing on similar gene therapies for sickle cell disease and for other bone marrow-derived diseases. (medicalxpress.com)
Gene therapy has long promised a solution for inherited diseases untreatable with conventional medicine. (imperial.ac.uk)
Gene-fixing treatments have now cured a number of patients with cancer and rare diseases. (technologyreview.com)
These results suggest that, with further work, this technique may keep HIV-infected patients free of disease symptoms," said the study's senior author, Wenzhe Ho, M.D., of the Division of Immunologic and Infectious Diseases at The Children's Hospital of Philadelphia. (sciencedaily.com)
The antitat gene offers the possibility of prolonging the latency period indefinitely without the need for long-term antiretroviral treatment," said Stuart E. Starr, M.D., chief of Immunologic and Infectious Diseases at The Children's Hospital of Philadelphia, and a co-author of the study. (sciencedaily.com)
Improvements in the technology of gene delivery identified by Arts and his colleagues could lead to many more effective studies that help patients with many different diseases, including cancer. (health.am)
Gene therapy is a powerful technology which allows for modification of person's genes in order to develop antibodies for treating or curating diseases. (medgadget.com)
Currently, more than 20 gene-therapy drugs/products have been approved in the clinic which is indicated for wide range of cancer, blood disorders, and autoimmune diseases. (medgadget.com)
Apart from this, several other gene therapy products have been granted orphan designation which is indicated for the management of rare diseases. (medgadget.com)
With the growing incidence of cancer and other targeted diseases such as genetic disorders, the adoption of gene therapy has significantly increased. (medgadget.com)
Meanwhile, gene therapies are in focus with several companies targeting to develop a one-time gene therapy-based treatment for diseases. (zacks.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. (whnt.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. (whnt.com)
The future of life-changing cures resides in CRISPR based (gene-editing) technology," said Dr. Helen O'Neill of University College London. (whnt.com)
The therapy was the second for this disease to use CRISPR-Cas9 technology and the first to target a new genetic area and use cryopreserved stem cells with the hope of increasing access to such a treatment. (medicalxpress.com)
A gene editing therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics works exceptionally well. (pharmavoice.com)
In early 2021, Tsogbe received an experimental transplant of his own stem cells, which had been collected and edited in a laboratory using CRISPR gene editing, biomedicine's most cutting-edge tool. (pharmavoice.com)
From the abstract: 'CRISPR-Cas9 disruption of the HBG1 and HBG2 gene promoters was an effective strategy for induction of fetal hemoglobin. (cdc.gov)

We took fish that had the otoferlin gene knocked out and looked at all of the genes, the entire genome, and compared them to the genes of a normal fish," Johnson said. (reachmd.com)
The concept of gene therapy is elegant: like computer bugs, faulty letters in the human genome can be edited and replaced with healthy ones. (singularityhub.com)
Because the in vitro potency of an LNP formulation rarely reflects its in vivo performance, they directly evaluated the delivery specificity and efficacy in mice that have a reporter gene in their cells that lights up red when genome editing occurs. (eurekalert.org)
Therefore, an expanded tool- kit of therapeutic modalities to include traditional biotherapeutics (i.e. genome editing, gene silencing, and proteins) will be required to integrate many of the next generation targets into drug discovery campaigns. (sbir.gov)

However, BioNTech's expertise expands beyond just mRNA disruptive therapies, with other areas exploring Chimeric antigen receptors (CAR-T) and T-cell receptor inhibitors (which they have already partnered on with Eli-Lilly) and general checkpoint inhibition using its UniCell Platform ( more details here ). (labiotech.eu)

The use of the word 'cure' in relation to sickle cell disease or thalassemia has, up until now, been incompatible," she said in a statement, calling the MHRA's approval of gene therapy "a positive moment in history. (whnt.com)
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. (whnt.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. (wave3.com)
Gene variants regulating fetal hemoglobin and a-thalassemia were frequently identified, but other associations remained unconfirmed. (cdc.gov)

With the help of follow-up immunohistochemistry, chromatin immunoprecipitation-qPCR, cytokine profiling, mouse modeling, and other experiments, the team suggested that Fusobacterium infection can boost transforming growth factor beta (TGF-beta) activation, prompting quiescent fibroblast cells to become myofibroblast cells expressing a cytoplasmic microfilament-associated protein known as transgelin (TAGLN). (genomeweb.com)
The results suggested that parenchymal cells that line the ventricles and surface of the brain took up the injected genes from the cerebrospinal fluid and made more TPP1 protein. (nih.gov)
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)
Attempts have been made to treat AAT deficiency by introducing the missing protein into patients' lungs (an approach called protein replacement therapy), but the results have not been encouraging. (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)
If you grow up without that protein, it's not just a matter of throwing the gene back in. (reachmd.com)
The genetic study showed promising results in "silencing" the genes that translate into tau protein, the primary component behind dementia. (medicaldaily.com)
What does this gene/protein do? (cancerindex.org)
What pathways are this gene/protein implicaed in? (cancerindex.org)
Scientists gave cancerous lab mice a gene therapy that turned off the production of the Myc protein, which causes tumor growth. (medicaldaily.com)
This contract focuses on gene therapy, which has the advantage over protein therapy to target specific cells for gene transduction leading to production or deduction of proteins precisely where therapy is needed. (sbir.gov)
The study suggests that the approach can enable the targeted delivery of a gene that encodes micro-dystrophin, a smaller version of the protein at the heart of DMD. (scienceboard.net)

Although further studies are needed, our data suggest that targeting Fusobacterium in the endometrium by antibiotic treatment may be a therapeutic option for patients with endometriosis," the authors concluded. (genomeweb.com)
LONDON (AP) - 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. (whnt.com)
According the article , there has been a new study involving gene therapy, that has been so successful that there have been no side effects with the treatment. (punnettssquare.com)
New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD). (medicalxpress.com)
In the larger context of therapeutic development, LaBelle stressed the importance of the study's contribution to the growing body of evidence supporting the viability of gene therapy as a treatment for sickle cell disease. (medicalxpress.com)
The treatment, however, did not fully improve the dogs' vision suggesting that delivery to the eye itself may be necessary as well. (nih.gov)
FRIDAY, June 30, 2023 (HealthDay News) -- The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A. The treatment, Roctavian (valoctocogene roxaparvovec), will cost $2.9 million for a single infusion, the Associated Press reported. (healthday.com)
Before now, treatment has involved FVIII replacement therapy or an antibody-based medication, the FDA said. (healthday.com)
The gene therapy treatment aims to deliver stable expression of the ATP7B copper transporter, which plays a role in the regulation of copper homeostasis in the liver by delivering copper to the secretory pathway and mediating export of excess copper into the bile, following a single intravenous infusion. (ucdavis.edu)
We are encouraged by the safe administration of the gene therapy and promising preliminary results, but the same success needs to be seen in more patients before the treatment can be approved," said Medici. (ucdavis.edu)
When doctors first tried it 20 years ago, the treatment had unintended effects on other genes, and some patients later developed leukemia. (nbcnews.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)
The therapy could be a safe and effective treatment for this rare and life-threatening condition. (nih.gov)
Gene therapy is a potential treatment option for infants and older children with ADA-SCID. (nih.gov)
These results suggest that gene therapy could be a safe and effective treatment for ADA-SCID. (nih.gov)
These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA-SCID," says NIAID Director Dr. Anthony S. Fauci. (nih.gov)
Our data demonstrate that HSV-TK/GCV suicide gene therapy system is effective for treating experimental human pancreatic cancer, which is largely resistant to the common therapies, so the suicide gene therapy system may be a potential treatment approach for pancreatic cancer. (wjgnet.com)
Over 20% of patients taking Nirmatrelvir-ritonavir therapy, commonly known as Paxlovid, suffer from the rebound, compared to less than the 2% chance seen in other treatment groups, a Harvard Medical School study revealed. (medicaldaily.com)
If so, the strategy could become an alternative to the current standard of AIDS treatment, highly active antiretroviral therapy (HAART). (sciencedaily.com)
At the turn of the century, the death of an 18-year- old patient with inherited liver disease after an experimental gene therapy treatment put the entire field into a deep freeze. (singularityhub.com)
The treatment uses a virus to insert a functional piece of DNA into the eyes to override a defective gene. (singularityhub.com)
Led by professor Eric Arts, PhD, the scientists discovered that the process of gene therapy is missing essential elements thereby reducing the effectiveness of this treatment. (health.am)
Advances in gene therapy offer promising treatment for genetic abnormalities, tumors and resistance to toxic chemotherapies. (health.am)
Yet treatment may not be as simple as its dramatic benefit suggests. (pharmavoice.com)
The challenge when altering the genetics of our cells, however, is how to do it safely, effectively, and specifically targeted to the gene, tissue and organ that needs treatment. (eurekalert.org)
For instance, in November 2021, Sio Gene Therapies reported positive interim data for gene therapy trial of Phase I/II of AXO-AAV-GM1 for the treatment of GM1 gangliosidosis, a genetic disorder that progressively destroys nerve cells in the brain and spinal cord. (medgadget.com)
Neuroprotective therapies would be a leap forward, meeting the needs of patients who lack treatment options. (newswise.com)
This newer avenue of treatment also has potential to earn high revenues as very few companies have been successful in developing an effective gene therapy. (zacks.com)
A new media panel, gene kit and advanced purification resins help reduce manufacturing costs while increasing the viability of gene therapies as treatment options for patients. (pharmiweb.com)
Gene therapy might offer a one-time, sustained treatment for patients with serious alcohol addiction, also called alcohol use disorder, according to a new study led by a researcher at The Ohio State University Wexner Medical Center and College of Medicine. (yalibnan.com)
Our findings suggest that this treatment can prevent relapse without requiring long-term treatment adherence by patients. (yalibnan.com)
Our focus is translational research that advances the understanding of cancer treatment resistance mechanisms through the development of novel targeted therapies in order to improve patient survival. (massgeneral.org)
Consequently, endocrine therapy, including tamoxifen or aromatase inhibitors (AIs), has become an effective treatment for these patients. (spandidos-publications.com)
In addition the beneficial uses of NPs in drug delivery, cancer treatment, and gene therapy may cause unintentional human exposure. (cdc.gov)
New research suggests that earlier and more effective treatment for Duchenne muscular dystrophy, an inherited life-limiting disorder caused by a genetic. (scienceboard.net)
Conclusion: Expression of ERh is an independent marker for favorable prognosis after adjuvant tamoxifen treatment in ERa-negative breast cancer patients and involves a gene expression program distinct from ERa. (lu.se)
Knowing whether or not you have the gene change may help your provider decide on your treatment plan. (medlineplus.gov)
In addition to targeted therapies a second generation of immune therapies has been developed with anti-PD1/PDL1 as the main choice of treatment. (lu.se)

This lentiviral vector was the platform technology licensed to Boehringer Ingelheim for further development of cystic fibrosis gene therapy in 2021. (imperial.ac.uk)
Such therapy has been used to treat cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia. (health.am)

For instance, US FDA has recently granted orphan drug designation to RGX-202, one-time experimental gene therapy for the management of Duchenne muscular dystrophy. (medgadget.com)

To support their findings, the scientists tested the significance of mEAK-7's role in cell proliferation and migration by inhibiting the gene in living human cells. (innovitaresearch.com)
In a study of dogs, scientists showed that a new way to deliver replacement genes may be effective at slowing the development of childhood Batten disease, a rare and fatal neurological disorder. (nih.gov)
Further inspections of the dogs' brains confirmed the scientists' hypothesis about how the therapy worked. (nih.gov)
A study out Wednesday details how scientists turned this enemy virus into a savior, altering it so it couldn't cause disease and then using it to deliver a gene the boys lacked. (nbcnews.com)
Scientists removed stem cells from the boy's bone marrow and modified them in the lab by introducing copies of a gene to prevent his red blood cells from becoming "sickled. (technologyreview.com)
While scientists continue looking for molecular therapies that may someday restore the hearing of those who carry the mutation, the research led by Colin Johnson of the OSU College of Science also has implications for improving the lives of people whose hearing can't come back. (reachmd.com)
Scientists at Tufts University and the Broad Institute of Harvard and MIT have developed unique nanoparticles comprised of lipids -- fat molecules -- that can package and deliver gene editing machinery specifically to the liver. (eurekalert.org)
Scientists from the International Agency for Research on Cancer (IARC) and international partners have discovered a potential mechanism underlying the roots of endocrine resistance that commonly develops in response to anti-estrogen therapy. (who.int)

ExperimentalDesign: We investigated ERh by immunohistochemistry in 353 stage II primary breast tumors from patients treated with 2 years adjuvant tamoxifen, and generated gene expres- sion profiles for a representative subset of 88 tumors. (lu.se)

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. (whnt.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. (whnt.com)
It involves removing some of a patient's blood cells, using the modified HIV to insert the missing gene, and returning the cells through an IV. (nbcnews.com)
This involves removing some of the patient's own stem cells, adding a normal copy of the ADA gene, and then returning the modified cells to the patient. (nih.gov)
Gene therapy, the introduction of genetic material into a patient's cells to rewrite genetic instructions and treat disease, is a young field in the early stages of potential applications. (sciencedaily.com)
But a failure to distribute enough modified genetic information to the patient's body has prohibited gene therapy from being more widely used. (health.am)
Gene therapy relies mainly on viruses-which transport genomes inside the cells they infect-to deliver genetic material into a patient's cells. (health.am)

Just before Christmas, the FDA dropped another bombshell with its approval of Luxturna , the first gene therapy that targets mutated DNA in a specific gene. (singularityhub.com)
The kit targets conserved regions of multiple kanamycin resistance gene families, covering all common plasmids with kanamycin resistant genes used in bioproduction for applications such as triple transfection of host cells to produce AAVs. (pharmiweb.com)
This gene-therapy approach targets changes in dopamine function in the brain's mesolimbic reward pathway that are caused by chronic alcohol use," says co-principal investigator and co-corresponding author Krystof Bankiewicz, MD, PhD, professor of Neurological Surgery and director of the Brain Health and Performance Center at Ohio State. (yalibnan.com)

The UK Respiratory Gene Therapy Consortium in 2021. (imperial.ac.uk)

With multiple CAR-T therapies going through the pipeline , 2018 may very likely welcome new members onto the gene therapy scene. (singularityhub.com)

Purpose: Endocrine therapies, such as tamoxifen, are commonly given to most patients with estrogen receptor (ERa) ^ positive breast carcinoma but are not indicated for persons with ERa-negative cancer. (lu.se)
Mrs. Berta Kamprad Foundation, John and practice as a marker associated with prognosis and response to Augusta Persson Foundation for Medical Science, and University Hospital of Lund endocrine therapies such as tamoxifen (4). (lu.se)
Finally, the study revealed a selective reversibility and persistence of epigenetic changes observed after estrogen deprivation and re-stimulation, suggesting a potential mechanism underlying the roots of endocrine resistance that develops in response to anti-estrogen therapy. (who.int)

We came up with a list of abnormally expressed genes - some in the hair cells, and there were also neuronal genes altered, genes involved in the growth and wiring of neurons. (reachmd.com)

Alpha Mannosidosis is a rare inherited metabolic storage disease caused by a mutation in the gene for alpha-mannosidase, an enzyme that normally breaks down sugars (carbohydrates) in lysosomes. (ssa.gov)
New and promising therapies for alpha mannosidosis include bone marrow transplantation, enzyme replacement, and gene therapy. (ssa.gov)
One cause of SCID is a mutation in the gene for an enzyme called adenosine deaminase (ADA). (nih.gov)
Only one U.S. patient and one U.K. patient had to resume enzyme-replacement therapy because the modified stem cells didn't take hold. (nih.gov)

Preclinical work carried out with the GTC's lentiviral vector suggests they can be administered repeatedly without problems. (imperial.ac.uk)
Delivery and success of gene therapy for human cells has the potential of increasing five to ten times with the introduction of the GRPE into the lentiviral vector. (health.am)

Decades in the making , gene therapy-the idea of modifying a person's DNA to treat disease-represents a major shift in medicine. (technologyreview.com)

These findings suggest that a lethal lung disease can be targeted and corrected in a human lung organoid model in vitro . (nature.com)
The findings suggest a way forward for developing neuroprotective therapies for glaucoma, a leading cause of visual impairment and blindness. (newswise.com)

BioMarin is one company working on a gene therapy that replaces the faulty gene involved in the most common type of hemophilia, effectively curing the disorder. (technologyreview.com)

In co-culture with human cortical neurons and after transplantation (AT) into human brain tissue resected from patients with drug-resistant epilepsy, light-activated channelrhodopsin-2 (ChR2) expressing hdINs induced postsynaptic currents in human neurons, strongly suggesting functional efferent synapse formation. (lu.se)

The expression of this gene is regulated by interferon-gamma and lipopolysaccharide. (cancerindex.org)
The very interesting data, however, related to the measurement of the tumor mutational burden (TMB) and the interferon gamma gene signature. (medscape.com)

T his suggests the existence of other specific mechanisms in the endometrium that facilitate the development of endometriosis in patients," Kondo explained, noting that "there is no conclusive explanation about this question so far. (genomeweb.com)
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. (whnt.com)
There's been a great deal of effort towards offering patients different types of transplants with decreased toxicities, and now gene therapy rounds out the set of available treatments, so every patient with sickle cell disease can get some sort of curative therapy if needed. (medicalxpress.com)
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)
Notably, all U.K. patients and 90% of U.S. patients were able to stop antibody-replacement therapy by the end of the studies. (nih.gov)
Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life. (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. (wave3.com)
Called CAR-T therapies, these "living drugs" are made by extracting T cells from patients and genetically engineering them to go after and destroy cancer cells. (technologyreview.com)
It's possible, Johnson added, that future gene therapies may be best targeted toward very young patients, whose brains have not undergone as complete a rewiring as those of adult patients. (reachmd.com)
A study conducted in cell cultures, headed by a research team at The Children's Hospital of Philadelphia, has shown that inserting a beneficial gene into blood immune cells taken from patients infected with HIV blocked the AIDS virus from replicating in those cells. (sciencedaily.com)
The therapy has been shown to delay or halt progressive blindness in patients, but the lack of long-term follow-up data makes it difficult to say whether the benefits can last decades. (singularityhub.com)
A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients. (health.am)
Enhanced gene therapy and other improvements in targeted cell delivery might eliminate the need for stem cells and allow cells to be administered directly into patients. (health.am)
Ultimately, our innovative solutions are developed to expedite the delivery of safer therapies to patients. (pharmiweb.com)
Patients receiving long-term transfusion therapy also require iron chelation. (medscape.com)
The current mode of administration that is typically used for brain delivery for gene therapy is the highly invasive intrathecal administration, while other methods such as intravenous injection would provide less physical burden on the patients. (sbir.gov)
This was a well-performed trial of adjuvant therapy with dabrafenib (BRAF inhibitor) plus trametinib (MEK inhibitor) in patients with resected stage IIIA, IIIB, and IIIC melanoma by the American Joint Committee on Cancer (AJCC) 7th edition criteria. (medscape.com)
The patients with BRAF amplification actually did a little bit better, but not statistically significantly so, than those without BRAF amplification when they received dabrafenib/trametinib (BRAF/MEK inhibitors) adjuvant therapy. (medscape.com)
TMB is probably a prognostic factor because patients with lower TMB seemed like they did a little bit better with dabrafenib and trametinib, suggesting that, unlike with immunotherapy, high TMB really doesn't yield benefit with dabrafenib and trametinib. (medscape.com)
Sequencing of genes of drug response in tumor DNA and implications for precision medicine in cancer patients. (cdc.gov)
CONTEXT: The waiting time for radioactive iodine therapy (WRAIT) after total thyroidectomy (TT) in patients with papillary thyroid cancer (PTC) and lymph node metastases (N1) has not been sufficiently investigated for risk of adverse outcomes. (bvsalud.org)
OBJECTIVE: This work aimed to estimate the effect of WRAIT on the outcomes of disease persistence and recurrence among patients with N1 PTC and investigate factors predictive of delayed radioactive iodine therapy (RAIT). (bvsalud.org)

Even though gene therapy is one of the most promising approaches to cure human hyper IgM syndrome, these results suggest that when we modify very tightly regulated genes such as cytokines or other growth factors, particular care has to be taken to avoid excessive stimulation of the target cells. (nki.nl)
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)
Microscopic image of cancer cells expressing the mEAK-7 gene, which was described in research led by UCLA's Dr. Paul Krebsbach. (innovitaresearch.com)
Once they identified mEAK-7 in human cells, they screened several types of human cells - including embryonic stem cells and fibroblasts, the cells that form connective tissues and aid in wound healing - to better understand how the gene worked. (innovitaresearch.com)
If we can find a way to control the duplication and migration of cells, including those responsible for human disease, we may be able to create opportunities for new therapies. (innovitaresearch.com)
The biggest take-home message is that there are now more potentially curative therapies for sickle cell disease than ever before that lie outside of using someone else's stem cells , which can bring a host of other complications," said James LaBelle, MD, Ph.D., director of the Pediatric Stem Cell and Cellular Therapy Program at UChicago Medicine and Comer Children's Hospital and senior author of the study. (medicalxpress.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)
They isolated skin stem cells and added copies of a healthy version of the gene. (technologyreview.com)
We then transferred the HSV-TK gene to target cells SW1990 using these progeny virions, and treated these gene-modified tumor cells with GCV to study the sensitivity of the cells to GCV and their bystander effects by routine MTT-method. (wjgnet.com)
In those cells, the antitat gene inhibited HIV activation and replication. (sciencedaily.com)
Additionally, the antitat gene prolonged the survival of immune system cells called CD4+ T lymphocytes, an essential part of the body's immune defenses and primary target cells for the AIDS virus. (sciencedaily.com)
In this way, the antitat gene therapy might contribute to maintaining HIV in a dormant state in latently infected cells. (sciencedaily.com)
However, recent studies have suggested that HAART alone will not lead to eradication of the virus in the infected cells. (sciencedaily.com)
Early indications are that the antitat gene does not affect uninfected cells or cause toxic side effects. (sciencedaily.com)
In 2017, a 44-year-old man became the first person to receive a gene-editing therapy that directly modifies his cells. (singularityhub.com)
Here, the therapy used an older gene-editing tool called zinc finger nucleases, which corrected a genetic error that throws the body's metabolism out of whack and slowly destroys its cells. (singularityhub.com)
The therapy is bespoke, created from an individual's own stem cells via a laborious and expensive process. (pharmavoice.com)
Exa-cel also comes with the theoretical risk that wayward gene edits in the transplanted cells may one day lead to other problems, like cancer. (pharmavoice.com)
Together with earlier studies demonstrating delivery to the brain or T cells suggest a future possibility of highly targeted gene editing therapies. (eurekalert.org)
Newswise - A form of gene therapy protects optic nerve cells and preserves vision in mouse models of glaucoma, according to research supported by NIH's National Eye Institute. (newswise.com)
Using an antibody marker of CaMKII activity, Chen's team discovered that CaMKII pathway signaling was compromised whenever retinal ganglion cells were exposed to toxins or trauma from a crush injury to the optic nerve, suggesting a correlation between CaMKII activity and retinal ganglion cell survival. (newswise.com)
Searching for ways to intervene, they found that activating the CaMKII pathway with gene therapy proved protective to the retinal ganglion cells. (newswise.com)
Administering the gene therapy to mice just prior to the toxic insult (which initiates rapid damage to the cells), and just after optic nerve crush (which causes slower damage), increased CaMKII activity and robustly protected retinal ganglion cells. (newswise.com)
Among gene therapy-treated mice, 77% of retinal ganglion cells survived 12 months after the toxic insult compared with 8% in control mice. (newswise.com)
Similarly, boosting CaMKII activity via gene therapy proved protective of retinal ganglion cells in glaucoma models based on elevated eye pressure or genetic deficiencies. (newswise.com)
AAV is a non-pathogenic virus with the ability to infect cells at various stages of growth, and has become a preferred "viral vector" for delivering gene therapies. (pharmiweb.com)
The vast majority of these cells did not divide, suggesting that the transgene was indeed regulated in a similar fashion as the endogenous GFAP gene. (lu.se)
Remarkably, many of the observed changes upon estrogen deprivation were also detected in breast cancer cells that developed resistance in response to anti-estrogen therapy. (who.int)

One-and-done gene therapy - Injecting a shot of genes into the brain's ventricular plumbing system may be an effective long term method for treating neurological disorders. (nih.gov)

Last year, Britain approved a gene therapy for a fatal genetic disorder that had a list price of £2.8 million ($3.5 million). (wave3.com)
Instead of just treating symptoms like the vast majority of drugs on the market, gene therapy aims to correct the underlying genetic cause of a disease. (technologyreview.com)
During virus production, GRPE coordinates the production and filling of the container with the genetic material of HIV-1, or the desired human gene. (health.am)
Your provider may suggest that you have genetic testing to look for the gene changes that raise the risk for ovarian cancer. (medlineplus.gov)

The company that makes the therapy, Spark Therapeutics, said it won't be announcing its price until January. (technologyreview.com)

Spinocerebellar ataxia type 4 was one of these, but now a research team in Lund, Sweden, have identified the gene that is responsible. (lu.se)
Now, a research team in Lund has finally pinpointed a mutation in the ZFHX3 gene as the cause of the disease. (lu.se)

NIH-funded animal study suggests one-shot approach to injecting genes. (nih.gov)
The study suggests the possibility of using trans-vaccenic acid found in red meat and dairy products as a nutritional supplement to complement treatments for cancer. (medicaldaily.com)
The study suggests this approach as a promising therapy for AUD, with key findings demonstrating a significant reduction in alcohol intake in treated subjects. (yalibnan.com)

New variants will pose a challenge, but early signs suggest the shots will still boost antibody responses. (technologyreview.com)
Alternatively spliced transcript variants have been identified for this gene. (cancerindex.org)

Dr. Davidson and her team undertook a highly innovative approach for Batten disease gene therapy," said Jill Morris, Ph.D., program director at NIH's National Institute of Neurological Disorders and Stroke. (nih.gov)
We believe this approach shows merit for further study as a promising therapy for AUD and possibly other substance-abuse disorders. (yalibnan.com)

Now 10 babies with " bubble boy disease " have had it fixed by a gene therapy made from one of the immune system's worst enemies - HIV, the virus that causes AIDS . (nbcnews.com)
Gene therapy restored immune system function to children with an inherited immune disorder with fewer side effects than existing therapies. (nih.gov)
Previously, the field of gene therapy was struggling with numerous challenges and barriers such as immune responses and non-target effects. (medgadget.com)
Previously, we have established that melanoma can be grouped in gene expression phenotypes with immune-rich melanomas having improved patient survival. (lu.se)
MITF-low Proliferative, MITF-high Pigmentation and High-immune response are the dominant ones suggesting two distinct lineages of melanoma (MITF-high and low), which is also supported by in vitro studies. (lu.se)

It reduces the risk of uncontrolled bleeding by delivering the gene into the liver, to increase blood levels of FVIII. (healthday.com)
This therapy delivers the gene to the liver to replace the copper transporter that is mutated and allows the liver to function properly so it can eliminate accumulated copper," explained Medici. (ucdavis.edu)
This constitutes a strong rationale for testing liver gene transfer as a therapeutic option for MSUD. (nature.com)
We just have to make sure we deliver the gene editing package specifically to the liver so as not to create unwanted side effects. (eurekalert.org)

Last year, we wrote that 2016 was gene therapy's most promising year . (technologyreview.com)

To investigate the possible effects of the expression of this tightly regulated gene in vivo, we produced transgenic mice in which CD40L expression was deregulated. (nki.nl)

We alter the deficiency by infecting the SFTPB deficient iPSCs with a lentivirus carrying the wild type SFTPB gene. (nature.com)
Using lentivirus for gene transfer appears to be a safe option," said Stanton L. Gerson, MD, director of the Case Comprehensive Cancer Center and the Asa and Patricia Shiverick- Jane Shiverick (Tripp) Professor of Hematological Oncology at Case Western Reserve School of Medicine and director of the Seidman Cancer Center at University Hospitals Case Medical Center, who is not involved in the study. (health.am)

With that in mind, he and his colleagues turned to gene expression profiling, single-cell RNA sequencing, and fluorescence in situ hybridization (FISH) to assess uterus endometrial fibroblast and ovarian endometriotic lesion samples from individuals with endometriosis and samples from unaffected controls. (genomeweb.com)
Other gene therapy studies for SCD have used lentiviruses-a type of virus often modified and used for gene editing which remain in the cell long-term. (medicalxpress.com)
We used a replication defective recombinant retrovirus vector GINaTK (bearing HSV-TK gene) to make packaging cell PA317 produce progeny virions. (wjgnet.com)
With solutions that span the entire LV workflow, we provide high-quality products and expertise to help companies develop breakthrough cell and gene therapies. (thermofisher.com)
According to the U.S. Centers for Disease Control and Prevention (CDC), "Sickle cell trait is not a disease, but having it means that a person has inherited the sickle cell gene from one of his or her parents. (cdc.gov)
If a child inherits two sickle cell genes, one from each parent, then they will have sickle cell disease. (cdc.gov)
In this regard, cell-based therapy may be an alternative therapeutic approach. (lu.se)

The easy-to-use Applied Biosystems resDNASEQ Quantitative Plasmid DNA - Kanamycin Resistance Gene Kit * offers an all-in-one solution for precise and rapid measurement of residual plasmid DNA. (pharmiweb.com)
Findings from the Cusack Laboratory have shown that conventional anti-cancer therapies, such as radiation and chemotherapy, induce resistance mechanisms under the control of the gene transcription factor NF-kappaB. (massgeneral.org)
However, the downside of targeted therapies is development of resistance. (lu.se)

The answer is yes, expression of some other genes is affected. (reachmd.com)
Adding otoferlin back in restored expression of those genes, but only partially. (reachmd.com)
Search the gene expression profiles from curated DataSets in the Gene Expression Omnibus (GEO) repository. (cancerindex.org)
An ERh gene expression profile was identified and was markedly different from the ERa signature. (lu.se)
The ability to regulate transgene expression will be crucial for development of gene therapy to the brain. (lu.se)
Using toxin-induced lesions we investigated to what extent transgene expression could be regulated in accordance with the activation of the endogenous GFAP gene. (lu.se)
An aspect of gene delivery to the brain that is currently being regulate the endogenous GFAP expression in a lesioned brain. (lu.se)
Transcriptome analysis revealed that estrogen deprivation leads to a global downregulation in gene expression, and more specifically of a gene involved in DNA demethylation ( TET2 ). (who.int)
In three consecutive papers we have firmly established the presence and significance of gene expression subtypes in melanoma. (lu.se)

This gene encodes a member of the cysteine-aspartic acid protease (caspase) family. (cancerindex.org)

The challenge of a gene therapy approach to AIDS is to choose the right viral genes to sabotage the AIDS virus. (sciencedaily.com)

At this time, there are no therapies that target circuits in the brain that are altered by sustained, heavy alcohol use," says co-principal investigator and co-corresponding author Kathleen Grant, PhD, chief and professor of Behavioral Neuroscience at the Oregon National Primate Research Center. (yalibnan.com)

NEW YORK - New research suggests bacteria may spur on the development of endometriosis, a condition marked by tissue growth outside of the endometrial cavity. (genomeweb.com)
The previous research uncovered a smaller version of otoferlin, a size that could be used in gene therapy. (reachmd.com)
The antitat research combines gene therapy with another promising approach in genetically based medicine: antisense therapy. (sciencedaily.com)
The key pharmaceutical companies are now focused on research and development sector pertaining to gene therapies. (medgadget.com)
The strong pipeline associated with the robust research and development activities suggests a positive future of gene therapy in pharmaceuticals market. (medgadget.com)
The major companies working in gene therapy market are Merck, Roche, Takeda Pharmaceutical, Novartis, Boehringer Ingelheim, Eli Lilly, and others which actively indulge in research and development activities. (medgadget.com)
Whether our customers are accelerating life sciences research, solving complex analytical challenges, improving patient diagnostics and therapies or increasing productivity in their laboratories, we are here to support them. (pharmiweb.com)

To better understand the nuances of these therapy types in immuno-oncology you can read our review of cancer treatments. (labiotech.eu)
Blocking these responses with targeted therapies augments the effectiveness of anti-cancer therapies. (massgeneral.org)

Anatomy20

Organisms35

Diseases46

Chemicals and Drugs70

Analytical, Diagnostic and Therapeutic Techniques and Equipment51

Phenomena and Processes35

Disciplines and Occupations2

Technology, Industry, Agriculture5

Humanities1

Information Science2

Health Care4

Adeno-assoc6

Mutations10

Researchers20

Clinical20

Sickle cell gene therapy1

Vectors9

Thermo Fisher Sci3

Diseases18

CRISPR7

Genome4

MRNA1

Thalassemia4

Protein12

Treatment38

Cystic fibrosis2

Experimental1

Scientists8

Tumors1

Patient's7

Targets3

20211

20181

Endocrine3

Neuronal1

Enzyme4

Lentiviral vector2

Person's1

Findings suggest2

Hemophilia1

Neurons1

Interferon-gamma2

Patients22

Cells28

Brain's1

Genetic4

Spark Therapeutics1

Lund2

Study suggests3

Variants2

Disorders2

Immune5

Liver4

Therapy's1

Mice1

Lentivirus2

Cell7

Resistance3

Expression9

Encodes1

AIDS1

Target1

Research7

Cancer2