Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome

Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome

Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is a monogenic disorder caused by mutations in the FOXP3 gene, required for generation of regulatory T (Treg) cells. Loss of Treg cells leads to immune dysregulation characterized by multi-organ autoimmunity and early mo...

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Bibliographic Details
Main Authors: Swati Singh, Cole M. Pugliano, Yuchi Honaker, Aidan Laird, M. Quinn DeGottardi, Ezra Lopez, Stefan Lachkar, Claire Stoffers, Karen Sommer, Iram F. Khan, David J. Rawlings
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
FOXP3
IPEX
hematopoietic stem cell
HSC editing
gene editing
CRISPR
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012300222X

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http://www.sciencedirect.com/science/article/pii/S232905012300222X

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