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Article

Optimizing Integration and Expression of Transgenic Bruton's Tyrosine Kinase for CRISPR-Cas9-Mediated Gene Editing of X-Linked Agammaglobulinemia.

The CRISPR journal - 1 Apr 2021

Gray David H, Villegas Isaac, Long Joseph, Santos Jasmine, Keir Alexandra, Abele Alison, Kuo Caroline Y, Kohn Donald B

Abstract excerpt

X-linked agammaglobulinemia (XLA) is a monogenic primary immune deficiency characterized by very low levels of immunoglobulins and greatly increased risks for recurrent and severe infections. Patients with XLA have a loss-of-function mutation in the Bruton's tyrosine kinase (BTK) gene and fail to produce mature B lymphocytes. Gene editing in the hematopoietic stem cells of XLA patients to correct or replace the...

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Identifiers

PMID: 33876953
DOI: 10.1089/crispr.2020.0080
PMCID: PMC8336228

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