Basic Information

Gene symbol HBB Synonyms CD113t-C, ECYT6, beta-globin Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description hemoglobin subunit beta

GTO ID GTC2008
Trial ID NCT03728322
Disease Thalassemia
Altered gene HBB
Therapeutic/Target gene Target gene
TherapyGene editing
Treatment HBB gene corrected iHSCs
PhaseEarly_Phase1
Recruitment statusUnknown
TitleA Safety and Efficacy Study of a Single Center, Open-label, Single Arm About the Gene Correction of HBB in Patient-specific iHSCs Using CRISPR/Cas9 That Intervent Subjests With β-thalassemia Mutations
Year2018
CountryChina
Company sponsorAllife Medical Science and Technology Co., Ltd.
Other ID(s)HBB HSC-01

Clinical Result

Cohort 1
Administration route intravenous infusion
Pts 12
Age Child, Adult

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