Gene therapy demonstrates reactivation of fetal hemoglobin synthesis in severe sickle cell disease, highlighting a nonviral curative strategy.
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region of
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Frangoul et al. (2024) studied this question.
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