Gene symbol | LDLR | Synonyms | FH, FHC, FHCL1, LDLCQ2 | Type of gene | protein-coding |
Chromosome | 19 | Map location | 19p13.2 | dbXrefs | |
Gene perturbation-related omics dataset | PerturbAtlas | PertOrg | ||||
Description | low density lipoprotein receptor |
GTO ID | GTC2180 |
Trial ID | NCT04080050 |
Disease | Familial Hypercholesterolemia |
Altered gene | LDLR |
Therapeutic/Target gene | Therapeutic gene |
Therapy | Gene transfer |
Treatment | RGX-501 |
Recruitment status | Active, Not Recruiting |
Title | A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 |
Year | 2019 |
Country | Canada|Netherlands|United States |
Company sponsor | REGENXBIO Inc. |
Other ID(s) | RGX-501-102 |
Vector information | |||||||||
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Cohort 1 | |||||||
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