| 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 | GTC3577 |
| Trial ID | NCT06024057 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | LX101 |
| Recruitment status | Not Recruiting |
| Title | An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy (LX101) in Patients With Leber's Congenital Amaurosis (LCA) |
| Year | 2023 |
| Country | China |
| Company sponsor | Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine |
| Other ID(s) | SHGH-LX101-EX |
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| Cohort 1 | |||||||
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