Modified lentiviral globin gene therapy for pediatric β

CD34(+) cell HSPC gene addition gene therapy globin insulator lentivirus red blood cell single-cell DNA sequencing β(0)/β(0) thalassemia

Journal

Cell stem cell
ISSN: 1875-9777
Titre abrégé: Cell Stem Cell
Pays: United States
ID NLM: 101311472

Informations de publication

Date de publication:
10 May 2024
Historique:
received: 28 07 2023
revised: 24 01 2024
accepted: 24 04 2024
medline: 18 5 2024
pubmed: 18 5 2024
entrez: 17 5 2024
Statut: aheadofprint

Résumé

β

Identifiants

DOI: 10.1016/j.stem.2024.04.021 PMID: 38759653
pubmed: 38759653
pii: S1934-5909(24)00175-9
doi: 10.1016/j.stem.2024.04.021
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Informations de copyright

Copyright © 2024 Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of interests Y.C. is a co-founder and advisor of BDgene Therapeutics. S. Ling and X.W. filed two patents related to this work (“Lentiviral vector applicable to gene therapy of thalassemia and sickle anemia,” ZL201910824134.8, September 2, 2019, China; and “Lentiviral vector applicable to gene therapy of thalassemia and sickle anemia,” ZL202210013838.9, January 6, 2022, China).

Auteurs

Shiqi Li (S)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Sikai Ling (S)

Key Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai 200240, China; BDgene Therapeutics, Shanghai 200240, China.

Dawei Wang (D)

Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.

Xiaoyuan Wang (X)

BDgene Therapeutics, Shanghai 200240, China.

Fangyuan Hao (F)

BDgene Therapeutics, Shanghai 200240, China.

Liufan Yin (L)

Sequanta Technologies, Shanghai 200131, China.

Zhongtao Yuan (Z)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Lin Liu (L)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Lin Zhang (L)

BDgene Therapeutics, Shanghai 200240, China.

Yu Li (Y)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Yingnian Chen (Y)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Le Luo (L)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Ying Dai (Y)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Lihua Zhang (L)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Lvzhe Chen (L)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China.

Dongjie Deng (D)

Sequanta Technologies, Shanghai 200131, China.

Wei Tang (W)

Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.

Sujiang Zhang (S)

Shanghai Institute of Hematology, State Key Laboratory of Medical Genomics, National Research Center for Translational Medicine at Shanghai, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China.

Sanbin Wang (S)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China, Kunming, Yunnan 650100, China. Electronic address: sanbin1011@163.com.

Yujia Cai (Y)

Key Laboratory of Systems Biomedicine (Ministry of Education), Shanghai Center for Systems Biomedicine, Shanghai Jiao Tong University, Shanghai 200240, China. Electronic address: yujia.cai@sjtu.edu.cn.

Classifications MeSH