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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Xehetasun bibliografikoak
Argitaratua izan da:Sci Rep
Egile Nagusiak: 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.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Nature Publishing Group UK 2019
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6616324/
https://ncbi.nlm.nih.gov/pubmed/31289301
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-45859-9
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