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 GTC1171
Trial ID NCT00004809
Disease Familial Hypercholesterolemia
Altered gene LDLR
Therapeutic/Target gene Therapeutic gene
TherapyGene transfer
Treatment rRTV-hLDLR
PhasePhase1
Recruitment statusCompleted
TitlePhase I Study of Ex Vivo Liver-Directed Gene Therapy for Familial Hypercholesterolemia
Year2000
CountryUnited States
Company sponsorNational Center for Research Resources (NCRR)
Other ID(s)199/12011|UPHS-28040
Vector information
Vectorretrovirus

Clinical Result

Cohort 1
Administration route infusion
Donor type autologous
Age Child, Adult, Older_Adult

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