November, 28, 2011 - Foundation-funded researchers from the University of Florida have taken a major step forward in the development of a vision-restoring, gene-replacement therapy for people with Leber congenital amaurosis (LCA) caused by mutations in the gene GUCY2D. They demonstrated that efficacy of the treatment in a mouse model of the condition was sustained for a year, a significant improvement over earlier studies in which the treatment’s effect lasted three months. The investigators believe that given these latest results, a similar treatment could last for several years or possibly a lifetime in humans.
越来越多的RP致病基因被攻克。 战友们，希望就在前方。 |
