Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

M. Maguire, Albert; Simonelli, Francesca; A. Pierce, Eric; N. Pugh, Edward; Mingozzi, Federico; Bennicelli, Jeannette; Banfi, Sandro; Marshall, Kathleen; Testa, Francesco; Maria Surace, Enrico; Rossi, Settimio; Lyubarsky, Arkady; R. Arruda, Valder; A. Konkle, Barbara; M. Stone, Edwin; Sun, Junwei; B. Jacobs, Jonathan; F Dell'Osso, Lou; W. Hertle, Richard; Ma, Jian‐xing; Michael Redmond, T.; Zhu, Xiaosong; Hauck, Bernd; Zelenaia, Olga; S. Shindler, Kenneth; G. Maguire, Maureen; Fraser Wright, J.; J. Volpe, Nicholas; Wellman McDonnell, Jennifer; Auricchio, Alberto; A. High, Katherine; Bennett, Jean

doi:10.1056/nejmoa0802315

Public

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Published in New England Journal of Medicine • Apr 27, 2008

Authors:

Albert M. Maguire

Francesca Simonelli

Eric A. Pierce

Abstract

Leber's congenital amaurosis (LCA) is a group of inherited blinding diseases with onset during childhood. One form of the disease, LCA2, is caused by mutations in the retinal pigment epithelium-specific 65-kDa protein gene (RPE65). We investigated the safety of subretinal delivery of a recombinant a...

Subject

Medicine

RPE65

Cis-trans-Isomerases

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