AAVs in Retinal Gene Therapy
Type
Blog Post
Published
July 31, 2018, 12:55 p.m.
...infecting cells of the retinal pigmented epithelium (RPE).
AAVs, on the other hand, have broad tropism in...Luckily for patients, this gene can be targeted to RPE cells using a lentiviral vector and a clinical trial...after the cloning of the RPE65 gene, a naturally occurring animal model, the RPE65−/− dog, was characterized...of several disease-causing genes, including the RPE65 gene responsible for an autosomal recessive, severe...study showing that AAV-mediated delivery of the RPE65 gene restores vision in the dog model of LCA (Lancelot...Lancelot, one of the dogs who received the AAV-RPE65 treatment, famously visited Congress to help increase...