AAVs in Retinal Gene Therapy
Type
Blog Post
... rare genetic eye disease, Leber’s congenital amaurosis. This is the first of what I, scientists, doctors...childhood-onset form of blindness name Leber’s Congenital Amaurosis (LCA). Shortly after the cloning of the RPE65 ...efficiently transduce various types of retinal neurons. Nowadays a toolbox of naturally occurring and ...muscular dystrophy, spinal muscular atrophy, neurodegenerative diseases and metabolic diseases are also underway...