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UConn Health University of Connecticut-Wesleyan University Stem Cell Core

Genome Editing 

Flow Chart for Gene Editing on hPSC

Genome editing will allow us to introduce specific genomic modification in hESCs/iPSCs, which is an essential tool to explore the full potential of stem cells in basic and translational research. Genome editing can be accomplished through the use of site-specific nucleases such as zinc finger nucleases (ZFNs), transcription activator-like effector nuclease (TALENs) or Cas9 directed by clustered regularly interspaced short palindromic repeats (CRISPRs). These nucleases will introduce site-specific double-strand DNA breaks. These breaks will then be repaired by either non-homologous end joint (NHEJ) or homologous recombination (HR). During NHEJ, the cells could introduce a small insertion or deletion (indel) at the site of the break leading to knockout of a gene if the break happens in the coding region of the gene. A more precise repair is through HR. In the presence of a specific donor vector, one could accomplish precise genetic insertion, deletion or replacement at the site of the DNA break. These genetic engineering strategie