AAVs in Retinal Gene Therapy
Type
Blog Post
...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...