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

Published: 18 April 2024| Version 1 | DOI: 10.17632/f9krjzw66k.1
Contributors:
Yujia Cai, Fangyuan Hao

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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Categories

Gene Therapy, Lentivirus, Beta Thalassemia

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

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