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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Foilsithe in:Gene Ther
Main Authors: 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
Formáid: Artigo
Teanga:Inglês
Foilsithe: Nature Publishing Group 2017
Ábhair:
Original Article
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC5398942/
https://ncbi.nlm.nih.gov/pubmed/28079862
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2017.4
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