| Gene symbol | RPE65 | Synonyms | BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65 | Type of gene | protein-coding |
| Chromosome | 1 | Map location | 1p31.3 | dbXrefs | |
| Description | retinoid isomerohydrolase RPE65 | ||||
| GTO ID | GTC1310 |
| Trial ID | NCT00821340 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | rAAV2-hRPE65 |
| Phase | Phase1 |
| Recruitment status | Completed |
| Title | Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-hRPE65) Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations |
| Year | 2009 |
| Country | Israel |
| Company sponsor | Hadassah Medical Organization |
| Other ID(s) | RPE65-HMO-CTIL |
| Vector information | |||||||||
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| Cohort 1 | |||||
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