Metabolite and thymocyte development defects in ADA-SCID mice receiving enzyme replacement therapy

Abstract Deficiency of adenosine deaminase (ADA, EC3.5.4.4), a housekeeping enzyme intrinsic to the purine salvage pathway, leads to severe combined immunodeficiency (SCID) both in humans and mice. Lack of ADA results in the intracellular accumulation of toxic metabolites which have effects on T cel...

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Autores principales: Federico A. Moretti, Giuliana Giardino, Teresa C. H. Attenborough, Athina Soragia Gkazi, Ben K. Margetts, Giancarlo la Marca, Lynette Fairbanks, Tessa Crompton, H. Bobby Gaspar
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/f65c7cd9d5074750a94e03ee0d0444ee
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