Modified Lentiviral Globin Gene Therapy for Pediatric β0/β0 Transfusion-Dependent β-Thalassemia: A Single-Center, Single-Arm Pilot Trial
Description
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.
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Funding
National Key Research and Development Program of China
2022YFC3400205
Science and Technology Innovation Action Plan of Shanghai
24Z510200306
National Natural Science Foundation of China
32370148
Key Forward-looking Fund from Shanghai Jiao Tong University
AF4150049
Biomedical Major Project of Yunnan Province
202102AA100011
Young and Middle-aged Academic and Technical Leaders Reserve Talent Project of Yunnan Province
202205AC160074