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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
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. 
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BigField GEG Tech's insight:
The authors describe methods for rapid synthesis of gRNA and for delivery of Cas9 protein/gRNA ribonucleoprotein complexes (Cas9 RNPs) into a variety of mammalian cells through liposome-mediated transfection or electroporation. Using these methods, they report nuclease-mediated indel rates of up to 94% in Jurkat T cells and 87% in induced pluripotent stem cells (iPSC) for a single target.
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The ability to reprogram adult somatic cells into induced pluripotent stem cells (iPSCs) and the subsequent development of protocols for their differentiation into disease-relevant cell types have enabled in-depth molecular analyses of multiple disease states as hitherto impossible.
BigField GEG Tech's insight:
Here the scientists demonstrate that CRISPRa and CRISPRi can be used to exert precise modulations of endogenous gene expression in fate-committed iPSC-derived neurons. This highlights CRISPRa/i as a major technical advancement in accessible tools for evaluating the specific contributions of critical neurodegenerative disease-related genes to neuropathogenesis.
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BigField GEG Tech's insight:
In this study, the scientists demonstrate that hPSC-derived cholangiocytes possess epithelial functions, including rhodamine efflux and CFTR-mediated fluid secretion. Furthermore, they show that functionally impaired hPSC-derived cholangiocytes from cystic fibrosis patients are rescued by CFTR correctors.
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BigField GEG Tech's insight:
In this study, the authors design high efficiency TALEN and ZFN to target two safe harbor sites on chromosome 13 and 19 to generate reporter systems while retaining pluripotent characteristics. They demonstrate the possibility to use a Cre-recombinase induced cassette exchange strategy to rapidly exchange reporter cassettes to develop new reporter lines in the same isogenic background at high efficiency. The results provide a novel platform for rapidly developing custom single or dual reporter systems for screening assays.
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To explore the impact of age on iPSC quality, the scientists produced iPSCs from blood cells of 16 donors aged 21–100. We find that iPSCs from older donors retain an epigenetic signature of age, which can be reduced through passaging. These studies establish that donor age is associated with an increased risk of abnormalities in iPSCs and will inform clinical development of reprogramming technology.