Gene Therapy in a Patient with Sickle Cell Disease
Abstract
Sickle cell disease results from a homozygous missense mutation in the β-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 antisickling β-globin gene into...
Paper Details
Title
Gene Therapy in a Patient with Sickle Cell Disease
Published Date
Mar 2, 2017
Volume
376
Issue
9
Pages
848 - 855
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