Commonalities 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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