Modified Lentiviral Globin Gene Therapy for Pediatric β0/β0 Transfusion-Dependent β-Thalassemia: A Single-Center, Single-Arm Pilot Trial

These data are the deposited supplemental information for the article entitled "Modified Lentiviral Globin Gene Therapy for Pediatric β0/β0 Transfusion-Dependent β-Thalassemia: A Single-Center, Single-Arm Pilot Trial" by Shiqi Li and Sikai Ling et al. in Cell Stem Cell.

Dataset 1. The insertion site information of WBCs from the two patients and their drug products obtained by next-generation sequencing.
Dataset 2. The impact of the HBBT87Q expression profile on PBMCs from four patient-derived samples at different time points after transplantation, assessed using single-cell RNA sequencing.