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Toward genome editing in X-linked RP—development of a mouse model with specific treatment relevant features
Genome editing represents a powerful tool to treat inherited disorders. Highly specific endonucleases induce a DNA double strand break near the mutant site, which is subsequently repaired by cellular DNA repair mechanisms that involve the presence of a wild type template DNA. In vivo applications of...
Autores principales: | , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Elsevier
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6294733/ https://www.ncbi.nlm.nih.gov/pubmed/30213530 http://dx.doi.org/10.1016/j.trsl.2018.08.006 |