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CRISPR-Cas9 used to correct sickle cell–causing mutation

August 07, 2018 / Russell Publishing Limited

Genome editing using the CRISPR-Cas9 system has tremendous promise for therapeutic correction of genetic errors in human cells. Prior to adoption as a medical therapeutic, safety concerns relating to off-target effects must be minimized to mitigate risks that may arise from the unintended action of this system at sites similar, but not identical, to the desired gene target site. Researchers at Integrated DNA Technologies (IDT) and the laboratory of Professor Matthew Porteus at Stanford University describe a novel Cas9 mutant that shows improved specificity and maintains high activity when used in the medically relevant ribonucleoprotein (RNP) format. The potential for medical use of the new mutant enzyme was demonstrated in the human hematopoietic stem and progenitor cells (HSPCs), where it was able to correct the mutation in the beta-hemoglobin gene responsible for sickle cell disease (SCD).