Yahoo HealthFirst-line immunotherapy treatment can improve survival for subset of lung cancer patientsMedical XpressFindings from a phase III clinical trial for advanced lung cancer patients could help oncologists better predict which patients are like… Continue reading
Regulatory FocusRegulating CRISPR: FDA and Industry Offer PerspectiveRegulatory FocusAlthough the US market is likely more than a year or two away from seeing any commercialized medical products that rely on CRISPR-Cas9 technology, the rapidly developi… Continue reading
IFLScience |
Scientists Reverse Huntington’s Disease In Mice Using CRISPR
IFLScience The research, published in the Journal of Clinical Investigation, focused on mice engineered to develop Huntington’s disease, with symptoms like impaired movement developing when they are nine months old. The team then used CRISPR to change the … |
|
New Project Aims to Set Effectiveness Guidelines for Hemophilia Gene Therapy Trials
Hemophilia News Today “With a growing pipeline of gene therapy products for hemophilia, it is an ideal time for this work,” Sean Tunis, president and CEO of CMTP, said in a news release. “This effort will potentially serve as a model for achieving consensus around outcomes … |
|
New mechanism for genome regulation discovered
Science Daily “Gene therapy, or any treatment that relies on tight regulation of gene expression, could be improved by precisely targeting molecules to the right place in the nucleus,” says Karpen. “It is very difficult to target genes located in heterochromatin … |
|
Stem Cells Try, But Fail, to Repair ACL Injuries
Newswise (press release) Newswise — The human body has a stockpile of stem cells, armed and ready to respond and repair damage to a variety of cells and tissues. Researchers have known heart attacks and strokes summon these cells, but, scientists recently discovered other … |
|
Sarepta and Genethon Collaborate on Gene Therapy Research for Duchenne
Rare Disease Report Unlike Sarepta’s current pipeline that is focused on specific dystrophin gene mutations, Genethon’s micro-dystrophin gene therapy approach can target most patients with DMD, and the company has demonstrated proof-of-concept via robust gene expression … |