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Article

Promise of gene therapy to treat sickle cell disease

16 Oct 2018

Abstract excerpt

INTRODUCTION: Sickle cell anemia (SCA) is a hereditary blood disease caused by a single-gene mutation that affects millions of individuals world-wide. In this review, we focus on techniques to treat SCA by ex vivo genetic manipulation of hematopoietic stem/progenitor cells (HSPC), emphasizing replacement gene therapy and gene editing. AREAS COVERED: Viral transduction of an anti-sickling β-like globin gene has...

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PMID: 30324810
DOI: 10.1080/14712598.2018.1536119

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