PRJNA839164
First 2 TDT patientis gene editing outcomes
This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with beta-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with gama-globin reactivated autologous hematopoietic stem cells in subjects with beta-thalassemia major. The next-generation sequencing data deposited here includes editing results in patient cells, as well as patient PBMC single-cell transcriptomes.