| 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 | GTC0315 |
| Trial ID | NCT02781480 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | AAV-RPE65 |
| Phase | Phase1|Phase2 |
| Recruitment status | Completed |
| Title | An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno Associated Virus Vector for Gene Therapy of Adults And Children With Retinal Dystrophy Associated With Defects in RPE65 (LCA) |
| Year | 2016 |
| Country | United Kingdom|United States |
| Company sponsor | MeiraGTx UK II Ltd |
| Other ID(s) | MGT003 |
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| Cohort1: dose level 1 | |||||||||||
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