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Article

Gene Therapy in a Patient with Sickle Cell Disease

1 Mar 2017

Abstract excerpt

Sickle cell disease results from a homozygous missense mutation in the -globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addition of an...

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PMID: 28249145
DOI: 10.1056/nejmoa1609677

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