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Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia

12 Aug 2025

Abstract excerpt

cDNA transgene directly into its endogenous locus. To study the effectiveness of this therapy, murine lineage-negative hematopoietic cells from a murine model of XLA were edited using CRISPR-Cas9/rAAV6 then transplanted into recipient XLA mice. Myeloablated XLA mice that received transplantation of Btk-corrected Lin- cells displayed high levels of engraftment, significant increases in their B cell levels,...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Identifiers

PMID: 40917692
DOI: 10.1016/j.omtm.2025.101555

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 5 Sept 2026.

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