Titlebook: Genome Editing; Kursad Turksen Book 2016 Springer International Publishing Switzerland 2016 CRISPR.ESC.iPSC.nucleases.stem cells

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Using CRISPR-Cas9 Genome Editing to Enhance Cell Based Therapies for the Treatment of Diabetes MellJ or via HDR. From the first demonstration that CRISPR-Cas can be programmed to cleave DNA in 2012, the field has advanced fast and now includes examples of targeting in many model organisms as well as gene knockout or reporter hPSC lines that will aid in the production of specific cell types, such as pancreatic β-cells.

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Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retiue as a viable treatment option for other inherited disorders across medical disciplines. This chapter highlights current ocular gene therapy approaches, clinical and preclinical experiments, and provides a case study of the bench-to-bedside personalized medicine approach taken for a novel and rare retinitis pigmentosa mutation.

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