Gene Therapy for Leber Congenital Amaurosis

  1. Gregory Bock Organizer,
  2. Gerry Chader Organizer and
  3. Jamie Goode
  1. Jean Bennett

Published Online: 7 OCT 2008

DOI: 10.1002/0470092645.ch15

Retinal Dystrophies: Functional Genomics to Gene Therapy: Novartis Foundation Symposium 255

Retinal Dystrophies: Functional Genomics to Gene Therapy: Novartis Foundation Symposium 255

How to Cite

Bennett, J. (2003) Gene Therapy for Leber Congenital Amaurosis, in Retinal Dystrophies: Functional Genomics to Gene Therapy: Novartis Foundation Symposium 255 (eds G. Bock, G. Chader and J. Goode), John Wiley & Sons, Ltd, Chichester, UK. doi: 10.1002/0470092645.ch15

Author Information

  1. FM Kirby Center for Molecular Ophthalmology, Scheie Eye Institute and Department of Ophthalmology, University of Pennsylvania, 310 Stellar-Chance Labs, 422 Curie Boulevard, Philadelphia PA 19104-2689, USA

Publication History

  1. Published Online: 7 OCT 2008
  2. Published Print: 9 DEC 2003

Book Series:

  1. Novartis Foundation Symposia

Book Series Editors:

  1. Novartis Foundation

ISBN Information

Print ISBN: 9780470853573

Online ISBN: 9780470092644

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Summary

Recent success in delivering vision to a canine model of a severe, early-onset blinding disease, Leber congenital amaurosis (LCA) (Acland et al 2001) demonstrates that adeno-associated virus serotype 2 (AAV2) is capable of delivering a corrective gene to the target retinal cells. Results of these studies indicate long-term rescue of vision as assessed by psychophysical, behavioural and molecular biological studies. Preliminary results of studies in progress are described and the implications of these results with respect to developing human clinical trials for LCA and for other retinal degenerative diseases are discussed.