| 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 | GTC0478 |
| Trial ID | NCT03913130 |
| Disease | Leber Congenital Amaurosis |
| Altered gene | CEP290 |
| Therapeutic/Target gene | Target gene |
| Therapy | ASO |
| Treatment | Sepofarsen|QR-110 |
| Phase | Phase1|Phase2 |
| Recruitment status | Terminated |
| Title | An Open-Label, Extension Study to Evaluate the Safety, Tolerability, Efficacy and Pharmacokinetics of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene |
| Year | 2019 |
| Country | united States|Belgium |
| Company sponsor | ProQR Therapeutics |
| Other ID(s) | PQ-110-002|2018-003500-40 |
| Vector information | |||
|
|||
| Cohort 1 | |||||||||
|
|||||||||