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Gene therapy for adenosine deaminase severe combined immune deficiency—An unexpected journey of four decades

30 Nov 2023

Abstract excerpt

Severe combined immune deficiency due to adenosine deaminase deficiency (ADA SCID) is an inborn error of immunity with pan-lymphopenia, due to accumulated cytotoxic adenine metabolites. ADA SCID has been treated using gene therapy with a normal human ADA gene added to autologous hematopoietic stem cells (HSC) for over 30 years. Iterative improvements in vector design, HSC processing methods, and clinical HSC...

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PMID: 38033164
DOI: 10.1111/imr.13293

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