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Gene therapy for severe combined immunodeficiencies and beyond

11 Dec 2019

Abstract excerpt

Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase (ADA) deficiency. The rationale was brought up by the observation of the revertant of SCIDX1 and ADA deficiency as a kind of natural gene therapy. Nevertheless, the first attempts of gene therapy for SCID X1 were associated with...

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PMID: 31826240
DOI: 10.1084/jem.20190607

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