A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

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A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

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Title: A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models
Author: Ding, Qiurong; Lee, Youn-Kyoung; Schaefer, Esperance Anne Kreek; Peters, Derek Tilghman; Veres, Adrian; Kim, Kevin; Kuperwasser, Nicolas; Motola, Daniel L; Meissner, Torsten B; Hendriks, William; Trevisan, Marta; Gupta, Rajat; Moisan, Annie; Banks, Eric; Friesen, Max; Schinzel, Robert T.; Xia, Fang; Tang, Alexander; Xia, Yulei; Figueroa, Emmanuel; Wann, Amy; Ahfeldt, Tim; Daheron, Laurence Marie; Zhang, Feng; Rubin, Lee; Peng, Lee F; Chung, Raymond Taeyong; Musunuru, Kiran; Cowan, Chad A.

Note: Order does not necessarily reflect citation order of authors.

Citation: Ding, Qiurong, Youn-Kyoung Lee, Esperance A.K. Schaefer, Derek T. Peters, Adrian Veres, Kevin Kim, Nicolas Kuperwasser, et al. 2013. “A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models.” Cell Stem Cell 12 (2) (February): 238–251. doi:10.1016/j.stem.2012.11.011.
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Abstract: Transcription activator-like effector nucleases (TALENs) are a new class of engineered nucleases that are easier to design to cleave at desired sites in a genome than previous types of nucleases. We report here the use of TALENs to rapidly and efficiently generate mutant alleles of 15 genes in cultured somatic cells or human pluripotent stem cells, the latter for which we differentiated both the targeted lines and isogenic control lines into various metabolic cell types. We demonstrate cell-autonomous phenotypes directly linked to disease—dyslipidemia, insulin resistance, hypoglycemia, lipodystrophy, motor-neuron death, and hepatitis C infection. We found little evidence of TALEN off-target effects, but each clonal line nevertheless harbors a significant number of unique mutations. Given the speed and ease with which we were able to derive and characterize these cell lines, we anticipate TALEN-mediated genome editing of human cells becoming a mainstay for the investigation of human biology and disease.
Published Version: doi:10.1016/j.stem.2012.11.011
Other Sources: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3570604/pdf/nihms423745.pdf
Terms of Use: This article is made available under the terms and conditions applicable to Open Access Policy Articles, as set forth at http://nrs.harvard.edu/urn-3:HUL.InstRepos:dash.current.terms-of-use#OAP
Citable link to this page: http://nrs.harvard.edu/urn-3:HUL.InstRepos:14068402
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