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Article

Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety.

Molecular therapy : the journal of the American Society of Gene Therapy - 7 Mar 2018

Cicalese Maria Pia, Ferrua Francesca, Castagnaro Laura, Rolfe Katie, De Boever Erika, Reinhardt Rickey R, Appleby Jonathan, Roncarolo Maria Grazia, Aiuti Alessandro

Abstract excerpt

Loss of adenosine deaminase activity leads to severe combined immunodeficiency (ADA-SCID); production and function of T, B, and natural killer (NK) cells are impaired. Gene therapy (GT) with an autologous CD34+-enriched cell fraction that contains CD34+ cells transduced with a retroviral vector encoding the human ADA cDNA sequence leads to immune reconstitution in most patients. Here, we report short- and...

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Source

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Identifiers

PMID: 29433935
DOI: 10.1016/j.ymthe.2017.12.022
PMCID: PMC5910668

Genes

ADA

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