| Gene symbol | USH2A | Synonyms | RP39, US2, USH2, dJ1111A8.1 | Type of gene | protein-coding |
| Chromosome | 1 | Map location | 1q41 | dbXrefs | |
| Description | usherin | ||||
| GTO ID | GTC0561 |
| Trial ID | NCT05085964 |
| Disease | Retinitis Pigmentosa |
| Altered gene | USH2A |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | QR-421a|ultevursen |
| Phase | Phase2 |
| Recruitment status | Terminated |
| Title | An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia) |
| Year | 2021 |
| Country | Canada|France|United States |
| Company sponsor | ProQR Therapeutics |
| Other ID(s) | PQ-421a-002|2021-002070-93 |
| Vector information | |||
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| Cohort1: dose level 1 | |||||||||||
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| Cohort2: dose level 2 | |||||||||||
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