Subset of stem cells identified as source for all cells in blood and immune...
Researchers at Fred Hutchinson Cancer Research Center have identified a specific subset of adult blood stem cells that is exclusively responsible for repopulating the entire blood and immune system...
View ArticlePhase 1 study shows encouraging data for gene replacement therapy for SMA type I
A one-time intravenous infusion of the high dose of gene therapy extended the survival of patients with spinal muscular atrophy type 1 (SMA1) in a Phase 1 clinical trial, according to a study published...
View ArticleNew gene therapy targets brain disorders
UNSW researchers have tailored gene therapy for a rare genetic disorder called Canavan disease, and say it could eventually be adapted to treat other inherited disorders affecting the brain.
View ArticleGenetic treatment for blindness may soon be reality
Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO...
View ArticleUS scientists try first gene editing in the body
Scientists for the first time have tried editing a gene inside the body in a bold attempt to permanently change a person's DNA to try to cure a disease.
View ArticleNew gene therapy transplantation technique could improve treatment of...
A therapeutic technique to transplant blood-forming (hematopoietic) stem cells directly into the brain could herald a revolution in our approach to treating central nervous system diseases and...
View ArticleOne-dose gene therapy produces clotting factor, safely stops bleeding in...
A team of gene therapy researchers has reported positive results in a phase 1/2 clinical trial for the inherited bleeding disorder hemophilia B. A single intravenous infusion of a novel bioengineered...
View ArticleGene therapy shows promise against blood-clotting disease
Gene therapy has freed 10 men from nearly all symptoms of hemophilia for a year so far, in a study that fuels hopes that a one-time treatment can give long-lasting help and perhaps even cure the blood...
View ArticleInflammatory factors linked to inhibition of factor VIII gene therapy in...
As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting...
View ArticleGene therapy improves immunity in babies with 'bubble boy' disease
Early evidence suggests that gene therapy developed at St. Jude Children's Research Hospital will lead to broad protection for infants with the devastating immune disorder X-linked severe combined...
View ArticleGroundbreaking gene therapy trial set to cure hemophilia
A 'cure' for haemophilia is one step closer, following results published in the New England Journal of Medcine of a groundbreaking gene therapy trial led by the NHS in London.
View ArticleThe story of a new skin
Decades of scientific research into areas including plant genetics and data science helped doctors successfully carry out an experimental therapy to create a new skin for a seven-year-old boy suffering...
View ArticleEarly diagnosis can save babies' lives: A guide to severe combined...
A new review provides guidance on a deadly, but rare, disease that is potentially curable if identified early. Severe combined immunodeficiency disease (SCID), known as the "bubble boy disease" in the...
View ArticleGene therapy for rare form of blindness wins US approval
U.S. health officials on Tuesday approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness. It marks another major...
View ArticleCommonalities in late stages of inherited blinding diseases suggest targets...
Gene therapy holds promise for treating a variety of diseases, including some inherited blinding conditions. But for a gene therapy to be effective, one must know the precise gene responsible for a...
View ArticleFirst step toward CRISPR cure of Lou Gehrig's disease
University of California, Berkeley scientists have for the first time used CRISPR-Cas9 gene editing to disable a defective gene that causes amyotrophic lateral sclerosis, or Lou Gehrig's disease, in...
View ArticleIn a milestone year, gene therapy finds a place in medicine
After decades of hope and high promise, this was the year scientists really showed they could doctor DNA to successfully treat diseases. Gene therapies to treat cancer and even pull off the...
View ArticleGene therapy restores normal blood glucose levels in mice with type 1 diabetes
Type 1 diabetes is a chronic disease in which the immune system attacks and destroys insulin-producing beta cells in the pancreas, resulting in high blood levels of glucose. A study published January...
View ArticleThe coming of age of gene therapy: A review of the past and path forward
After three decades of hopes tempered by setbacks, gene therapy—the process of treating a disease by modifying a person's DNA—is no longer the future of medicine, but is part of the present-day...
View ArticleCancer's gene-determined 'immune landscape' dictates progression of prostate...
The field of immunotherapy - the harnessing of patients' own immune systems to fend off cancer - is revolutionizing cancer treatment today. However, clinical trials often show marked improvements in...
View ArticleGene delivery of drugs directly into arthritic joints is making the leap to...
Localized gene delivery to diseased joints to achieve sustained drug production at the site of osteoarthritis or rheumatoid arthritis is gaining momentum, with clinical trials underway in the U.S. and...
View ArticleResearchers identify gene responsible for mesenchymal stem cells' stem-ness'
Many doctors, researchers and patients are eager to take advantage of the promise of stem cell therapies to heal damaged tissues and replace dysfunctional cells. Hundreds of ongoing clinical trials are...
View ArticleDiscovery may advance neural stem cell treatments for brain disorders
New research from Sanford Burnham Prebys Medical Discovery Institute (SBP) is among the first to describe how an mRNA modification impacts the life of neural stem cells (NSCs). The study, published in...
View ArticleViral gene therapy could improve results from breast reconstruction after...
A new type of gene therapy delivered using a virus could protect healthy tissues from the harmful side-effects of radiotherapy after cancer treatment, a new study reports.
View ArticleGene-modified, virus-specific T cell therapy shows promise in treating...
In a first-of-its-kind clinical study, Baylor College of Medicine evaluated the safety, survival and anti-tumor activity of tumor-specific T cells rendered resistant to transforming growth factor β...
View ArticleResearchers cure lung fibrosis in mice with a gene therapy that lengthens...
Idiopathic pulmonary fibrosis is a potentially lethal disease associated with the presence of critically short telomeres, currently lacking effective treatment. The Telomere and Telomerase Group at the...
View ArticleNew study shows severe toxicity with high-dose AAV9-based gene therapy in...
Researchers have described severe, life-threatening toxicity in monkeys and piglets given high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector capable of accessing spinal...
View ArticleGene therapy researchers find viral barcode to cross the blood-brain barrier
Gene therapies promise to revolutionize the treatment of many diseases, including neurological diseases such as ALS. But the small viruses that deliver therapeutic genes can have adverse side effects...
View ArticleStudy sheds new light on mechanism of breast cancer treatment resistance
A study by researchers at Dana-Farber Cancer Institute has illuminated a specific mechanism by which estrogen receptor-positive (ER+) breast cancers can become resistant to standard therapies and...
View ArticleCan gene therapy be harnessed to fight the AIDS virus?
For more than a decade, the strongest AIDS drugs could not fully control Matt Chappell's HIV infection. Now his body controls it by itself, and researchers are trying to perfect the gene editing that...
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