This is a single centre、single arm、open-label study,to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Study Type
INTERVENTIONAL
Purpose
TREATMENT
Masking
NONE
Enrollment
12
iHSCs intravenous injection
Occurrence of treatment related adverse events as assessed by CTCAE v4.0
Defined as \>= Grade 3 signs/symptoms, laboratory toxicities, and clinical events) that are possibly, likely, or definitely related to study treatment
Time frame: 1 year
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