| 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 | GTC0585 |
| Trial ID | 2015-003418-25 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | AAV2/5-OPTIRPE65 |
| Phase | Phase1 |
| Recruitment status | Completed |
| Title | An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with Defects in RPE65 (LCA2) |
| Year | 2016 |
| Country | United Kingdom |
| Company sponsor | MeiraGTx UK II Ltd |
| Vector information | |||||||||||
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| Cohort 1 | |||||||||||||
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