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Recombinase-Mediated Reprogramming and Dystrophin Gene Addition in mdx Mouse Induced Pluripotent Stem Cells

Zhao, Chunli, Farruggio, Alfonso P., Bjornson, Christopher R. R., Chavez, Christopher L., Geisinger, Jonathan M., Neal, Tawny L., Karow, Marisa, Calos, Michele P.·2014·PLoS ONE
DOI 10.1371/journal.pone.0096279PMC PMC4004573
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  • Advances in identifying safe harbor loci in the human genome have facilitated in vitro transgene expression for large genes, such as human dystrophin and beta-hexosaminidases in Tay-Sachs and Sandhoff's syndromes, as well as expression of multiple glycosyl hydrolases in the CEP112 locus in animal models.

    “advances in identifying safe harbor loci in the human genome have facilitated in vitro transgene expression for large genes, such as the human dystrophin [169] and beta-hexosaminidases in Tay-Sachs and Sandhoff’s syndromes [170], as well as the expression of multiple glycosyl hydrolases in the CEP112 locus in animal models [171].”

    From The Mitochondrial Genome in Aging and Disease and the Future of Mitochondrial Therapeutics (2022) · cites this paper as [169]

    The Results (Addition of dystrophin cDNA to mdx iPSC; page unavailable) report integration of a full-length dystrophin transgene at a selected safe site, and the Discussion reports dystrophin expression in the engineered cells. But the transgene was mouse dystrophin cDNA in mouse iPSC, not human dystrophin at a safe-harbor locus in the human genome. The other genes in the citing sentence have separate citations and are not attributed to this paper. (LLM, conf=0.90)

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Research works printed but not linked: 44

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Metadata

Authors
Zhao, C., Farruggio, A. P., Bjornson, C. R. R., Chavez, C. L., Geisinger, J. M., Neal, T. L., Karow, M., & Calos, M. P.
Venue
PLoS ONE
Publisher
Public Library of Science (PLoS)
Published
2014-04-29
Volume
9
Issue
4
Pages
e96279
ISSN
1932-6203
DOI
10.1371/journal.pone.0096279
PMC
PMC4004573
Cited by
25
References
2
Language
en
Licence
creativecommons.org/licenses/by/4.0/ (unspecified)

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