In an article published in the April 8 issue of Nature, the National Institutes of Health’s Somatic Cell Gene Editing Consortium provided a detailed update on the progress of their nationwide effort to develop safer and more effective methods to edit the genomes of disease-relevant somatic cells and reduce the burden of disease caused by… Read More »
Transplanted cells persist for 19 months in man with HIV – but don’t appear to control HIV when treatment stops CRISPR gene editing was safe and moderately effective in introducing stem cells that lacked the CCR5 receptor and were immune to HIV infection after chemotherapy eradicated the immune system of a man with HIV and… Read More »
FDA Approves Zolgensma (onasemnogene abeparvovec-xioi) Gene Therapy to Treat Pediatric Patients with Spinal Muscular Atrophy Print this page May 24, 2019 — The U.S. Food and Drug Administration today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe… Read More »
(Reuters) – Novartis AG, which this week announced positive interim trial results for its experimental gene therapy for spinal muscular atrophy, on Friday said investigation is underway into whether a second trial death could be related to the treatment. The logo of Swiss drugmaker Novartis AG is seen at its headquarters in Basel, Switzerland January… Read More »