Correcting the Genetic Error in Sickle Cell Disease

Editing of sickle hematopoetic stem/progenitor cells reduces sickle (hbS) and increases normal (hbA) hemoglobin.

The CRISPR/Cas9 DNA editing system offers the potential for revolutionary new treatments of genetic diseases. Pioneering work by Carroll set the stage for this revolution by characterizing how cells detect and repair double-stranded DNA breaks. Sickle cell disease is common among African Americans and widespread in tropical regions of Africa and southern Asia.

Carroll and his colleagues used the CRISPR/Cas9 gene editing system and a novel delivery method to correct the sickle mutation in adult human hematopoietic stem/progenitor cells. Continuing work on this approach has improved the efficiency of correction, and treatment of the first patients is anticipated by the end of 2020.

Dana Carroll, PhD. Photo credit: Charlie Ehlert

References:

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Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells. DeWitt MA, Magis W, Bray NL, Wang T, Berman JR, Urbinati F, Heo SJ, Mitros T, Muñoz DP, Boffelli D, Kohn DB, Walters MC, Carroll D, Martin DI, Corn JE. Science Translational Medicine. 2016 Oct;8(360):360ra134.

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