Basic Information

Gene symbol LDLR Synonyms FH, FHC, FHCL1, LDLCQ2 Type of gene protein-coding
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
TherapyGene transfer
Treatment RGX-501
Recruitment statusActive, Not Recruiting
TitleA Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
Year2019
CountryCanada|Netherlands|United States
Company sponsorREGENXBIO Inc.
Other ID(s)RGX-501-102
Vector information
Vectoradeno-associated virus
ConstructAAV8-hLDLR
Vector typeadeno-associated virus serotype 8 (AAV8) vector
Transgene/Inserted genea functional copy of the human low-density lipoprotein receptor (LDLR) gene

Clinical Result

Cohort 1
Administration route intravenous infusion
Age Adult, Older_Adult
References : REGENXBIO Announces Initiation of Phase I/II Clinical Trial of RGX-501 for the Treatment of Homozygous Familial Hypercholesterolemia

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