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Gene-targeting and Animal Model Unit, iPSC and Genome Engineering Core, Center for Molecular Medicine, NHLBI

Friday, November 08, 2013 — Poster Session III

10:00 a.m. – 12:00 p.m.

FAES Academic Center (Upper-Level Terrace)




  • Y. Du
  • W. Xie
  • C. Liu


The NHLBI Gene-targeting and Animal Model Unit was established in October, 2012. It major responsibilities include: 1) generating transgenic and knockout/knockin mouse models; 2) editing the genome of ESCs and iPSCs through various genome-engineering methods; 3) deriving and characterizing non-human ESCs and iPSCs as well as developing animal models to support stem cell research. Our expertise in precisely modifying the genome of ESCs and microinjecting early embryos has traditionally enabled us to generate transgenic and knockout mouse models. In recent years, these skills have been increasingly used to support ESC/iPSC research, including editing the genome of iPSC lines and assessing stem cell’s differentiation potential in vivo. Conversely, some of the new technologies developed primarily for stem cell research, including ZFN, Talen, and CRISPR/Cas methods, have substantially shorten the timeline and increased the efficiency for creating genetically engineered mouse models. Furthermore, these new methods largely eliminated the species barrier, and made it possible to generate gene-targeted models in essentially any species. Our unit is aimed at taking advantage of the synergies between the animal model field and the stem cell field to develop capabilities that complement the expertise possessed by the other members of the iPSC and Genome Engineering Core.

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