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

ABSTRACT: 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 develop a gene correction strategy potentially applicable to most WAS patients by employing nuclease-mediated, site-specific integration of a corrective WAS gene sequence into the endogenous WAS chromosomal locus. In this study, we demonstrate the ability to target the integration of WAS_2-12-containing constructs into intron 1 of the endogenous WAS gene of primary CD34⁺ hematopoietic stem and progenitor cells (HSPCs), as well as

SUBMITTER: Pille M 

PROVIDER: S-EPMC10897892 | biostudies-literature | 2024 Mar

REPOSITORIES: biostudies-literature