Gene therapy will soon become the dominant modality for treating of sickle cell disease (SCD). Currently, three technologies are the most promising: expression of transgenic globin genes via a lentiviral vector, controlled mutation of the β-globin control cluster by transgenic CRISPR-based ribonucleoprotein, and suppression of BCL11a mRNA by shRNA. In this review, we discuss the mechanism of each technology and how they correct the SCD pathology at the molecular level. We conclude by discussing potential directions future therapy may take.
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| http://dx.doi.org/10.1016/j.transci.2022.103566 | DOI Listing |
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