AAVs in Retinal Gene Therapy
Type
Blog Post
Published
July 31, 2018, 12:55 p.m.
... used successfully in preclinical and clinical settings for ocular conditions. However, AdV was abandoned...responsible for an autosomal recessive, severe, childhood-onset form of blindness name Leber’s Congenital Amaurosis... of new capsid variants, designing expression cassettes to regulate and optimize transgene levels, and...