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ADA-SCID Gene Therapy Endorsed By European Medicines Agency For Marketing Authorization

1 Jun 2016

Abstract excerpt

The Committee for Human Medicinal Products has recently recommended marketing authorization for retrovirus-mediated ex vivo gene therapy of patients' autologous CD34+ cells transduced with human ADA complementary DNA for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).1 If confirmed by the European Commission, the product, called Strimvelis, would be the third approved gene...

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PMID: 27324442
DOI: 10.1038/mt.2016.98

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