Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Simonelli, Francesca; M. Maguire, Albert; Testa, Francesco; A. Pierce, Eric; Mingozzi, Federico; Bennicelli, Jeannette; Rossi, Settimio; Marshall, Kathleen; Banfi, Sandro; Maria Surace, Enrico; Sun, Junwei; Michael Redmond, T.; Zhu, Xiaosong; S. Shindler, Kenneth; Ying, Gui‐Shuang; Ziviello, Carmela; Acerra, Carmela; Fraser Wright, J.; Wellman McDonnell, Jennifer; A. High, Katherine; Bennett, Jean; Auricchio, Alberto

doi:10.1038/mt.2009.277

Public

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

Published in Molecular Therapy • Dec 1, 2009

Authors:

Francesca Simonelli

Albert M. Maguire

Francesco Testa

Abstract

The safety and efficacy of gene therapy for inherited retinal diseases is being tested in humans affected with Leber's congenital amaurosis (LCA), an autosomal recessive blinding disease. Three independent studies have provided evidence that the subretinal administration of adeno-associated viral (A...

Subject

Genetic enhancement

Administration (probate law)

Vector (molecular biology)

Research Assistant

AI chat, annotations, notes & similar papers

Finding related papers...

Discussions

(0)

No comments yet

Be the first to share your thoughts!