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Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency

26 Mar 2017

Abstract excerpt

BACKGROUND: Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase-deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (RIC) and ERT cessation. Clinical safety and therapeutic efficacy were evaluated in a phase II study. METHODS:...

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PMID: 28346229
DOI: 10.1172/jci90367

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