NeuroscienceMolecular TherapyPeer-reviewed
Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
Gustavo David Aguirre, Artur V. Cideciyan, Valérie Dufour…·William A. Beltran
This paper demonstrates that gene therapy using AAV-mediated NPHP5 augmentation restores photoreceptor structure and function in a canine model of Leber congenital amaurosis.