Journal
MOLECULAR THERAPY
Volume 25, Issue 5, Pages 1076-1094Publisher
CELL PRESS
DOI: 10.1016/j.ymthe.2017.03.008
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Funding
- Foundation Fighting Blindness-CHOP-Penn Pediatric Center for Retinal Degenerations, National Eye Institute/NIH grants [R21EY020662, 8DP1EY023177]
- Research to Prevent Blindness
- Brenda and Matthew Shapiro Stewardship
- Robert and Susan Heidenberg Investigative Research Fund for Ocular Gene Therapy
- Paul and Evanina Mackall Foundation Trust
- Center for Advanced Retinal and Ocular Therapeutics
- F.M. Kirby Foundation
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Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one earlyonset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.
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