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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...
Gorde:
| Argitaratua izan da: | Proc Natl Acad Sci U S A |
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| Egile Nagusiak: | , , |
| Formatua: | Artigo |
| Hizkuntza: | Inglês |
| Argitaratua: |
National Academy of Sciences
2016
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| Gaiak: | |
| Sarrera elektronikoa: | 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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