Researchers have developed a protocol that enables zinc-finger nuclease-mediated targeted editing of DNA sequences in human long-term repopulating haematopoietic stem cells (HSCs). Genovese et al. were able to successfully integrate a corrective complementary DNA downstream of the IL2RG (interleukin-2 receptor, gamma) promoter in blood-cell precursors from a patient with X-linked severe combined immunodeficiency syndrome, which is caused by mutations in IL2RG. Gene correction occurred in 3–11% of treated cells (depending on primitive versus committed progenitor status).
References
Genovese, P. et al. Targeted genome editing in human repopulating haematopoietic stem cells. Nature http://dx.doi.org/10.1038/nature13420 (2014)
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Koch, L. Genome editing in haematopoietic stem cells. Nat Rev Genet 15, 442 (2014). https://doi.org/10.1038/nrg3773
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DOI: https://doi.org/10.1038/nrg3773
