Preliminary findings indicate gene therapy pioneered at St. Jude Children's Research Hospital is safe and effective for babies with a devastating inherited disorder that leaves them with little or no immune protection.
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 factor VIII from the transferred FVIII gene.
Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer Inc. (NYSE:PFE), today announced that The New England Journal of Medicine has published interim data as of July 25, 2017, from the Phase 1/2 clinical trial of SPK-9001, an investigational gene therapy for hemophilia B.
Salk scientists redesign CRISPR to create precision medicine tool that does not damage DNA.
In a paper appearing in the journal Cell on Dec. 7, scientists at the Salk Institute report a modified CRISPR-Cas9 technique that alters the activity, rather than the underlying sequence, of disease-associated genes. The researchers demonstrate that this technique can be used in mice to treat several different diseases.
The technique, which could be used to transplant donor-matched hematopoietic stem cells (HSCs) or a patient's own genetically-engineered HSCs into the brain, was reported in Science Advances today by researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the San Raffaele Telethon Institute for Gene Therapy.
Researchers from Case Western Reserve University School of Medicine have discovered how unusually long pieces of RNA work in skin cells. The RNA pieces, called 'long non-coding RNAs' or 'lncRNAs,' help skin cells modulate connective tissue proteins, like collagen, and could represent novel therapeutic targets to promote skin repair.
A study published today in Science Advances shows for the first time the efficacy of a new gene therapy transplantation technique which aims at repopulating the brain with new, genetically engineered immune cells.
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 gene therapy enabled adult participants to safely produce sustained levels of clotting factor that prevented debilitating bleeding episodes. Patients were able to terminate prophylactic treatments; the gene therapy nearly universally eliminated their need for intravenous infusions of manufactured clotting factor.
You are what you eat when it comes to fat, report scientists from McGovern Medical School at The University of Texas Health Science Center at Houston (UTHealth) in the journal Science Advances.