Gene editing using CRISPR-Cas9 may allow targeted treatment for a variety of genetic diseases. These include inherited abnormalities of β hemoglobin, which can be indirectly targeted by increasing the amount of healthy fetal hemoglobin without fully correcting the disease-causing mutation. Humbert et al. used CRISPR-based gene editing to modify hematopoietic stem cells from nonhuman primates, introducing a naturally occurring mutation that increases the amount of fetal hemoglobin. The authors successfully applied this method to a highly enriched population of stem cells in the primate model, suggesting the potential for translating this efficient editing technique to human patients.


Sci. Transl. Med. 11, eaaw3768 (2019).



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