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Site-specific genome editing for correction of induced pluripotent stem cells derived from dominant dystrophic epidermolysis bullosa

Genome editing with engineered site-specific endonucleases involves nonhomologous end-joining, leading to reading frame disruption. The approach is applicable to dominant negative disorders, which can be treated simply by knocking out the mutant allele, while leaving the normal allele intact. We app...

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Bibliografske podrobnosti
izdano v:Proc Natl Acad Sci U S A
Main Authors: Shinkuma, Satoru, Guo, Zongyou, Christiano, Angela M.
Format: Artigo
Jezik:Inglês
Izdano: National Academy of Sciences 2016
Teme:
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC4878469/
https://ncbi.nlm.nih.gov/pubmed/27143720
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1512028113
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