Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
Children's Hospital of Philadelphia · Penn Presbyterian Medical Center · +11 more institutions
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Abstract
No abstract available for this paper.
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857
total citations
- FWCI
- 30.94
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- 100%
- References
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Authors
33- AMAlbert M. MaguireCorresponding
Children's Hospital of Philadelphia, Penn Presbyterian Medical Center, University of Pennsylvania
- KAKatherine A. High
Children's Hospital of Philadelphia, Howard Hughes Medical Institute, University of Pennsylvania, Philadelphia University
- AAAlberto Auricchio
Telethon Institute Of Genetics And Medicine
- JFJ. Fraser Wright
Children's Hospital of Philadelphia, University of Pennsylvania
- EAEric A. Pierce
Children's Hospital of Philadelphia, Penn Presbyterian Medical Center, University of Pennsylvania
Topics & keywords
Topics
Keywords
- RPE65
- Medicine
- Choroideremia
- Retinal pigment epithelium
- Genetic enhancement
- Retinal
- Ophthalmology
- Nystagmus
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