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CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges

Demirci, Selami, Leonard, Alexis, Haro-Mora, Juan J., Uchida, Naoya, Tisdale, John F.·2019·Advances in Experimental Medicine and Biology
DOI 10.1007/5584_2018_331
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Abstract

Sickle cell disease (SCD) is an inherited monogenic disorder resulting in serious mortality and morbidity worldwide. Although the disease was characterized more than a century ago, there are only two FDA approved medications to lessen disease severity, and a definitive cure available to all patients with SCD is lacking. Rapid and substantial progress in genome editing approaches have proven valuable as a curative option given plausibility to either correct the underlying mutation in patient-derived hematopoietic stem/progenitor cells (HSPCs), induce fetal hemoglobin expression to circumvent sickling of red blood cells (RBCs), or create corrected induced pluripotent stem cells (iPSCs) among other approaches. Recent discovery of CRISPR/Cas9 has not only revolutionized genome engineering but has also brought the possibility of translating these concepts into a clinically meaningful reality. Here we summarize genome engineering applications using CRISPR/Cas9, addressing challenges and future perspectives of CRISPR/Cas9 as a curative option for SCD.

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Metadata

Authors
Demirci, S., Leonard, A., Haro-Mora, J. J., Uchida, N., & Tisdale, J. F.
Venue
Advances in Experimental Medicine and Biology
Publisher
Springer International Publishing
Published
2019
Pages
37-52
ISSN
  • 0065-2598
  • 2214-8019
DOI
10.1007/5584_2018_331
Cited by
50
References
31
Language
en
Licence
www.springer.com/tdm (tdm)

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Demirci, S., Leonard, A., Haro-Mora, J. J., Uchida, N., & Tisdale, J. F. (2019). CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges. Advances in Experimental Medicine and Biology. https://doi.org/10.1007/5584_2018_331