Abstract

Mini Review

CRISPR9 SCD Gene Therapy

Mohammed Al Thani*

Published: 22 August, 2025 | Volume 9 - Issue 1 | Pages: 011-014

The most prevalent monogenic blood illness is sickle cell disease (SCD), which affects millions of people globally and around 100,000 Americans.

Read Full Article HTML DOI: 10.29328/journal.jhcr.1001035 Cite this Article Read Full Article PDF

References

  1. Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, et al. CRISPR-Cas9 gene editing for sickle cell disease and β-thalassemia. N Engl J Med. 2021;384(3):252-260. Available from: https://doi.org/10.1056/nejmoa2031054
  2. Ye L, Wang J, Tan Y, Beyer AI, Xie F, Muench MO, et al. Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia. Proc Natl Acad Sci U S A. 2016;113(38):10661-10665. Available from: https://doi.org/10.1073/pnas.1612075113
  3. Demirci S, Leonard A, Haro-Mora JJ, Uchida N, Tisdale JF. CRISPR/Cas9 for sickle cell disease: applications, future possibilities, and challenges. Cell Biol Transl Med. 2019;5:37-52. Available from: https://doi.org/10.1007/5584_2018_331
  4. Park SH, Lee CM, Deshmukh H, Bao G. Therapeutic CRISPR/Cas9 Genome Editing for treating sickle cell disease. Blood. 2016;128(22):4703. Available from: https://doi.org/10.1016/j.transci.2021.103060

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