Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Bainbridge, James; Mehat, Manjit; Sundaram, Venki; Robbie, Scott; E. Barker, Susie; Ripamonti, Caterina; Georgiadis, Anastasios; M. Mowat, Freya; G. Beattie, Stuart; J. Gardner, Peter; L. Feathers, Kecia; Luong, Vy; Yzer, Suzanne; S. Balaggan, Kamaljit; C. Viswanathan, Ananth; de Ravel, Thomy; Casteels, Ingele; E. Holder, Graham; Tyler, Nick; W. Fitzke, Fred; G. Weleber, Richard; Nardini, Marko; T. Moore, Anthony; A. Thompson, Debra; M. Petersen‐Jones, Simon; Michaelides, Michel; Ingeborgh van den Born, L.; Stockman, Andrew; J. Smith, Alexander; S. Rubin, Gary; R. Ali, Robin

doi:10.1056/nejmoa1414221

Public

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

Published in New England Journal of Medicine • May 4, 2015

Authors:

James Bainbridge

Manjit Mehat

Venki Sundaram

Abstract

BACKGROUND: Mutations in RPE65 cause Leber's congenital amaurosis, a progressive retinal degenerative disease that severely impairs sight in children. Gene therapy can result in modest improvements in night vision, but knowledge of its efficacy in humans is limited. METHODS: We performed a phase 1-2...

Subject

RPE65

Medicine

Genetic enhancement

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