CNS AAV AAV VECTORS FOR RETINAL AND CNS GENE THERAPY

摘要

An improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for improved gene therapy of ocular disorders or CNS disorders is provided herein, wherein the rAAV comprises one or more substitutions of amino acids that interact with heparan sulfate proteoglycans. The present invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases using improved compositions of rAAV particles. Improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for improved gene therapy of disorders of the CNS is further provided herein. The present invention provides methods for delivering rAAV to the CNS, methods for treating disorders of the CNS using improved compositions of rAAV particles, and kits for delivering rAAV and / or treating CNS disorders to the CNS.