| Gene symbol | CEP290 | Synonyms | 3H11Ag, BBS14, CT87, JBTS5, LCA10, MKS4, NPHP6, POC3, SLSN6, rd16 | Type of gene | protein-coding |
| Chromosome | 12 | Map location | 12q21.32 | dbXrefs | |
| Gene perturbation-related omics dataset | PertOrg | ||||
| Description | centrosomal protein 290 | ||||
| GTO ID | GTC0371 |
| Trial ID | NCT03140969 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | CEP290 |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | Sepofarsen|QR-110 |
| Phase | Phase1|Phase2 |
| Recruitment status | Completed |
| Title | An Open-label, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene |
| Year | 2017 |
| Country | Belgium|United States |
| Company sponsor | ProQR Therapeutics |
| Other ID(s) | PQ-110-001|2017-000813-22 |
| Vector information | |||
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| Cohort1: dose level 1 | |||||||||||||
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