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.

Categories

Funders

• Ministry of Science and Technology of the People's Republic of China

  China

  Grant ID: 2022YFC3400205
• Science and Technology Innovation Action Plan of Shanghai

  Grant ID: 24Z510200306
• National Natural Science Foundation of China

  China

  Grant ID: 32370148
• Key Forward-looking Fund from Shanghai Jiao Tong University

  Grant ID: AF4150049
• Biomedical Major Project of Yunnan Province

  Grant ID: 202102AA100011
• Young and Middle-aged Academic and Technical Leaders Reserve Talent Project of Yunnan Province

  Grant ID: 202205AC160074

Licence