NIH R01 · 2025
Engineering AAV for safe and efficient gene delivery to the human retina
ABSTRACT This is a competitive renewal of R01EY024280, “Engineering AAV for safe and efficient gene delivery to the human retina”. FDA approval of an Adeno associated virus (AAV)- based gene therapy for RPE65-Leber congenital amaurosis (LCA2) solidified gene therapy’s place in current medical practice. However, in the 6 years since, there have been no additional approvals, and multiple hurdles associated with retinal gene therapy have come to light. The development of therapies that safely and efficiently target the human retina remains a significant, unmet need. In previous funding cycles, we asked questions grounded in both basic and translational science. We have i) elaborated upon…
From the public funding record at NIH RePORTER. Describes the funded project, not the reviews below.