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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy

  • Published:2019-09 Issue:9 Volume:126 Page:1273-1285
  • ISSN:0161-6420
  • Container-title:Ophthalmology
  • language:en
  • Short-container-title:Ophthalmology

Author:

Maguire Albert M.,Russell Stephen,Wellman Jennifer A.,Chung Daniel C.,Yu Zi-Fan,Tillman AmyORCID,Wittes Janet,Pappas Julie,Elci Okan,Marshall Kathleen A.,McCague Sarah,Reichert Hannah,Davis Maria,Simonelli Francesca,Leroy Bart P.,Wright J. Fraser,High Katherine A.,Bennett Jean

Funder

Center for Cellular and Molecular Therapeutics

The Children's Hospital of Philadelphia

CHOP-Penn Pediatric Center for Retinal Degenerations

National Eye Institute

National Institutes of Health

Clinical Translational Science

NIH

National Center for Research Resources

National Center for Advancing Translational Sciences

Research to Prevent Blindness

Macula Vision Foundation

Paul and Evanina Mackall Foundation Trust

Publisher

Elsevier BV

Subject

Ophthalmology

Reference25 articles.

1. Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or Leber congenital amaurosis;Morimura;Proc Natl Acad Sci U S A,1998

2. Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration;Thompson;Invest Ophthalmol Vis Sci,2000

3. Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle;Redmond;Nat Genet,1998

4. Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle;Redmond;Proc Natl Acad Sci U S A,2005

5. Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy;Cideciyan;Prog Retin Eye Res,2010
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