Article
Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats
1 Apr 2016
Abstract excerpt
β -globinopathies (sickle-cell anemia and β-thalassemia) are the most common monogenic disorders worldwide that are potentially amenable to gene therapy. Proof-of-concept studies done 15 years ago, which showed correction of murine models of hemoglobinopathies, have gone through the rigors of preclinical safety and efficacy testing, and are now showing clinical promise both in β-thalassemia and sickle-cell...
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