Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease

Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease

X-linked severe combined immunodeficiency disease (X-SCID) is a rare inherited disease caused by mutations in the interleukin 2 receptor subunit gamma gene (IL2RG), which encodes the common γ chain protein, a subunit of the receptor for lymphocytes. X-SCID is characterized by profound defects in T-c...

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Main Authors: Mingfeng Hu, Qiling Xu, Fang Zhang, Karen F. Buckland, Yelei Gao, Weixia Du, Yuan Ding, Lina Zhou, Xiulian Sun, Lijia Ma, Zhiyong Zhang, Xuemei Tang, Xiaodong Zhao, Adrian J. Thrasher, Yunfei An
Format: Article
Language:English
Published: KeAi Communications Co., Ltd. 2025-05-01
Series:Genes and Diseases
Subjects:
Atypicaldiverse phenotype
Gene therapy
IL2RG
Self-inactivating lentiviral vector
X-linked severe combined immunodeficiency disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2352304224002423
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