Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness-Reference-Cited by-同舟云学术

Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness

Published:2010-03-03 Issue:21 Volume:2 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Amado Defne¹,Mingozzi Federico²,Hui Daniel²,Bennicelli Jeannette L.¹,Wei Zhangyong¹,Chen Yifeng²³,Bote Erin⁴,Grant Rebecca L.⁴,Golden Jeffrey A.⁵⁶,Narfstrom Kristina⁷,Syed Nasreen A.⁸,Orlin Stephen E.⁹,High Katherine A.²³,Maguire Albert M.¹²,Bennett Jean¹²

Affiliation:

1. F. M. Kirby Center for Molecular Ophthalmology, Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA.

2. Center for Cellular and Molecular Therapeutics, Children’s Hospital of Philadelphia, Abramson Pediatric Research Center, Philadelphia, PA 19014, USA.

3. Howard Hughes Medical Institute, Philadelphia, PA 19104, USA.

4. Nonhuman Primate Research Program, University of Pennsylvania, Department of Pathology and Laboratory Medicine, Gene Therapy Program, Translational Research Laboratories, Philadelphia, PA 19104-3402, USA.

5. Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.

6. Developmental Biology Program for Pediatric Disorders, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA.

7. College of Veterinary Medicine and Mason Eye Institute, University of Missouri-Columbia, Columbia, MO 65212, USA.

8. Departments of Ophthalmology and Visual Sciences and Pathology, University of Iowa, Iowa City, IA 52242-1007, USA.

9. Scheie Eye Institute, University of Pennsylvania, Philadelphia, PA 19104, USA.

Abstract

After successful gene therapy to correct retinal degeneration in one eye, treatment of the second eye is safe and effective—even when immunity to the vector is present—in nonhuman primates and dogs.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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