Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis
Gustavo David Aguirre, Artur V. Cideciyan, Valérie Dufour, Ana Ripolles‐Garcia, Raghavi Sudharsan, Małgorzata Świder, Roman Nikonov, Simone Iwabe, Sanford L. Boye, William W. Hauswirth, Samuel G. Jacobson, 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.
Molecular Therapy · Mar 27, 2021Peer-reviewedNeuroscience