Summary: Utilizing specialized adeno-associated viral (AAV) vectors to deliver functional human FMR1 directly into the central nervous system, the team successfully restored FMRP production within key cortical and subcortical regions…
Tag: Gene therapy
Gene Therapy Restores Hearing in Those with Deafness
Summary: A new study shows that gene therapy can significantly improve hearing in both children and adults with congenital deafness caused by mutations in the OTOF gene. The therapy uses…
MicroRNA is the Nobel-winning master regulator of the genome – researchers are learning to treat disease by harnessing how it controls genes
When Victor Ambros and Gary Ruvkun discovered a new molecule they called microRNA in the 1980s, it was a fascinating diversion from what for decades had been called the central…
