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Article

Twenty-Five Years of Gene Therapy for ADA-SCID: From <i>Bubble Babies</i> to an Approved Drug

29 Aug 2017

Abstract excerpt

Twenty-five years have passed since first attempts of gene therapy (GT) in children affected by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect, also known by the general public as bubble babies. ADA-SCID is fatal early in life if untreated. Unconditioned hematopoietic stem cell (HSC) transplant from matched sibling donor represents a curative treatment but is available for few...

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PMID: 28847159
DOI: 10.1089/hum.2017.175

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