CRISPR/Cas9 mediated generation of an ovine model for infantile neuronal ceroid lipofuscinosis (CLN1 disease)

The neuronal ceroid lipofuscinoses (NCLs) are a group of devastating monogenetic lysosomal disorders that affect children and young adults with no cure or effective treatment currently available. One of the more severe infantile forms of the disease (INCL or CLN1 disease) is due to mutations in the...

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Detalles Bibliográficos
Autores principales: Eaton, S. L., Proudfoot, C., Lillico, S. G., Skehel, P., Kline, R. A., Hamer, K., Rzechorzek, N. M., Clutton, E., Gregson, R., King, T., O’Neill, C. A., Cooper, J. D., Thompson, G., Whitelaw, C. B., Wishart, T. M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6616324/
https://www.ncbi.nlm.nih.gov/pubmed/31289301
http://dx.doi.org/10.1038/s41598-019-45859-9

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