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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Bibliografske podrobnosti
izdano v:Sci Rep
Main Authors: 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.
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group UK 2019
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Article
Online dostop: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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