All Entries Tagged With: "gene transfer"

Safe and efficient gene transfer approach ameliorated light perception

Leber’s Congenital Amaurosis (LCA) is a genetically inherited desease of the photoreceptors in the human retina. The patients loose light perception in their third decade of life due to a mutation in the protein retinal pigment epithelium–specific 65-kDa protein gene (RPE65) (Albert M. Maguire et al, 2008 NEJM). [PubMed] The consortium of institutions performing this [...]