| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 4029662 | Ophthalmology | 2007 | 4 Pages |
Abstract
The RPGRIP1-LCA patient has treatment potential for a gene replacement strategy if targeted to central, but not pericentral or peripheral, retina. The results differ from similarly studied RPE65-LCA and CRB1-LCA patients. Preclinical progress toward therapy in LCA patients warrants detailed structure-function studies in humans to determine feasibility and candidacy for clinical trials.
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Authors
Samuel G. MD, PhD, Artur V. PhD, Tomas S. MD, Alexander PhD, Sharon B. MS, CGC, Alejandro J. MS, Edwin M. MD, PhD,