For Leber hereditary optic neuropathy associated with an ND4 mutation: NR082 is an ophthalmic AAV-based gene therapy. It uses an AAV2 vector to express the human ND4 gene in the retinal ganglion cells to repair optic neuropathy caused by the mutation. Investigator-initiated trials began in 2011, which demonstrated the long-term safety, effectiveness, and durability of AAV gene therapy in […]
Click here to view original web page at www.scienceboard.net