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Genetic Engineering Publications - GEG Tech top picks
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Human pluripotent stem cells represent a unique source for cell-based therapies and regenerative medicine. The intrinsic features of these cells su...
BigField GEG Tech's insight:
This review offers a summary of the advanced methods recently developed to derive muscle progenitors from pluripotent stem cells, as well as gene therapy by gene addition and gene editing methods using ZFNs, TALENs or CRISPR/Cas9. The authors also discuss the main issues that need to be addressed for successful clinical translation of genetically corrected patient-specific pluripotent stem cells in autologous transplantation trials for skeletal muscle disorders. 
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BigField GEG Tech's insight:
In this study, the author compare ZFN and TALEN to target the insertion of a myelo-specific gp91phox cassette to AAVS1. They observe that TALENs were more efficient than ZFNs in generating correctly targeted iPSC colonies, but all corrected iPSC clones showed no signs of mutations at the top-ten predicted off-target sites of both nucleases. These data open the way to generate autologous, functionally corrected granulocytes with the TALEN system.
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BigField GEG Tech's insight:
This review gives a short primer on gene editing followed by some of the foundational work in gene editing and subsequently a discussion of programmable nucleases leading to a description of Zinc Finger Nuclease, TALENs and CRISPRs.
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This review presents the mechanisms of different gene editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, TALE nucleases, meganucleases, and the CRISPR/Cas9 system. The authors summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders.