Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis

Gustavo D. 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