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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 (...
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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BlackWell Publishing Ltd
2014
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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 |
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