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

CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges. Adv Exp Med Biol. 2019 Feb 05;: Authors: Demirci S, Leonard A, Haro-Mora JJ, Uchida N, Tisdale JF 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. PMID: 30715679 [PubMed - as supplied by publisher]
Source: Advances in Experimental Medicine and Biology - Category: Research Tags: Adv Exp Med Biol Source Type: research