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Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression

CLN2 neuronal ceroid lipofuscinosis is a hereditary lysosomal storage disease with primarily neurological signs that results from mutations in TPP1, which encodes the lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Studies using a canine model for this disorder demonstrated that delivery of TPP1 en...

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Detalles Bibliográficos
Autores principales:	Katz, M L, Johnson, G C, Leach, S B, Williamson, B G, Coates, J R, Whiting, R E H, Vansteenkiste, D P, Whitney, M S
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5398942/ https://www.ncbi.nlm.nih.gov/pubmed/28079862 http://dx.doi.org/10.1038/gt.2017.4

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