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

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

Abstract 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 mutation...

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Autores principales: S. L. Eaton, C. Proudfoot, S. G. Lillico, P. Skehel, R. A. Kline, K. Hamer, N. M. Rzechorzek, E. Clutton, R. Gregson, T. King, C. A. O’Neill, J. D. Cooper, G. Thompson, C. B. Whitelaw, T. M. Wishart
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2019
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Medicine
R
Science
Q
Acceso en línea:https://doaj.org/article/7155c59c589e4aa29b872c1109c35b0c
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https://doaj.org/article/7155c59c589e4aa29b872c1109c35b0c

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