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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...

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Detalles Bibliográficos
Autores principales:	Schlegel, J., Hoffmann, J., Röll, D., Müller, B., Günther, S., Zhang, W., Janise, A., Vössing, C., Fühler, B, Neidhardt, J., Khanna, H., Lorenz, B., Stieger, K.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Elsevier 2019
Materias:	Article
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

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