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Genetic Engineering Publications - GEG Tech top picks
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An international, peer-reviewed genome sciences journal featuring outstanding original research that offers novel insights into the biology of all organisms
BigField GEG Tech's insight:
The authors combined CRISPR/Cas9 technology with the piggyBac transposon to efficiently correct the HBB mutations in patient-derived iPSCs without leaving any residual footprint. No off-target effects were detected in the corrected iPSCs, and the cells retain full pluripotency and exhibit normal karyotypes. When differentiated into erythroblasts gene-corrected iPSCs restored expression of HBB compared to the parental iPSCs line.
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In this study, the scientists used the CRISPR system to do gene correction in reprogrammed fibroblasts of a patient with β-thalassemia into transgene-free naïve iPSCs with molecular signatures of ground-state pluripotency. Therefore, their findings demonstrate the feasibility and superiority of using patient-specific iPSCs in the naïve state for disease modeling, gene editing, and future clinical therapy.