The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
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Frequency and severity of adverse events(AEs) as assessed by CTCAE v5.0
Timeframe: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of engraftment
Timeframe: within 42 days post-CS-101 infusion
Time to neutrophil and platelet engraftment
Timeframe: Days post-CS-101 infusion
Occurrence of transplant-related death
Timeframe: baseline to 100 days post-CS-101 infusion
Occurrence of all-cause death
Timeframe: From signing informed consent to 180 days post-CS-101 infusion
Occurrence of achieving transfusion reduction for at least 3 consecutive months
Timeframe: From 3 months post -CS-101 infusion to 3 months post -CS-101 infusion