Article

CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells

2023-11-08

Abstract excerpt

<h4>ABSTRACT</h4> Interleukin 7 Receptor α Severe Combined Immunodeficiency (IL7R-SCID) is a life-threatening disorder caused by homozygous mutations in the IL7RA gene. Defective IL7R expression in humans hampers T cell precursors proliferation and differentiation during lymphopoiesis resulting in absence of T cells in newborns, who succumb to severe infections and death early after birth. Previous attempts to t...

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Literature Corpus work: 2ba3b4ac-22b8-5c12-816b-8929602dd8b2
DOI: 10.1101/2023.11.07.566043

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