Article
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia
11 Mar 2024
Abstract excerpt
BACKGROUND: X-linked agammaglobulinemia (XLA) is an inborn error of immunity that renders boys susceptible to life-threatening infections due to loss of mature B cells and circulating immunoglobulins. It is caused by defects in the gene encoding the Bruton tyrosine kinase (BTK) that mediates the maturation of B cells in the bone marrow and their activation in the periphery. This paper reports on a gene editing...
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