iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

Summary

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.

Timeline

Start

2019-01

Primary completion

2020-01

Completion

2021-01

Drugs

Indications