Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome

Wiskott-Aldrich syndrome (WAS) is a severe X-linked primary immunodeficiency resulting from a diversity of mutations distributed across all 12 exons of the WAS gene. WAS encodes a hematopoietic-specific and developmentally regulated cytoplasmic protein (WASp). The objective of this study was to deve...

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Bibliographic Details
Main Authors: Melissa Pille, John M. Avila, So Hyun Park, Cuong Q. Le, Haipeng Xue, Filomeen Haerynck, Lavanya Saxena, Ciaran Lee, Elizabeth J. Shpall, Gang Bao, Bart Vandekerckhove, Brian R. Davis
Format: Article
Language:English
Published: Elsevier 2024-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
gene editing
gene therapy
primary immune disease
Wiskott-Aldrich syndrome
immunology
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012400024X

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