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Genetic Engineering Publications - GEG Tech top picks
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BigField GEG Tech's insight:
A unique mouse model of Graves' disease, an autoimmune disorder that causes hyperthyroidism, has been developed by scientists, and new research findings may help improve the treatment of Graves' disease, experts report. 
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Scoop.it!
BigField GEG Tech's insight:
In this study, the authors report successful generation of human induced pluripotent stem cells (iPSC) from skin fibroblasts of a patient harboring a novel Ser331Cysfs*5 mutation in the MERTK gene. Upon differentiation of these iPSC towards RPE, patient-specific RPE cells exhibited defective phagocytosis, a characteristic phenotype of MERTK deficiency observed in human patients and animal models. This cellular model allows to investigate in detail the disease mechanism, explore screening of a variety of therapeutic compounds/reagents and design either combined cell and gene- based therapies or independent approaches.
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In this study, the scientists show that the in vitro application of an HDR enhancer, RS-1, increases the knock-in efficiency by two- to five-fold at different loci, whereas NHEJ inhibitor SCR7 has minimal effects. We then apply RS-1 for animal production and have achieved multifold improvement on the knock-in rates as well. This work presents tools to nuclease-mediated knock-in animal production, and sheds light on improving gene-targeting efficiencies on pluripotent stem cells.