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Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)

Using a canine model of classical late-infantile neuronal ceroid lipofuscinosis (CLN2 disease), a study was conducted to evaluate the potential pharmacological activity of recombinant human tripeptidyl peptidase-1 (rhTPP1) enzyme replacement therapy administered directly to the cerebrospinal fluid (...

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Detalles Bibliográficos
Autores principales: Katz, Martin L, Coates, Joan R, Sibigtroth, Christine M, Taylor, Jacob D, Carpentier, Melissa, Young, Whitney M, Wininger, Fred A, Kennedy, Derek, Vuillemenot, Brian R, O'Neill, Charles A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BlackWell Publishing Ltd 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4263309/
https://www.ncbi.nlm.nih.gov/pubmed/24938720
http://dx.doi.org/10.1002/jnr.23423