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Characterization of the molecular defect in a feline model for type II GM2-gangliosidosis (Sandhoff disease).

The Korat cat provides an animal model for type II GM2-gangliosidosis (Sandhoff disease) that may be suitable for tests of gene replacement therapy with the HEXB gene encoding the beta subunit of the beta-hexosaminidases. In the present report, we examined the brain and liver pathology of a typical...

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Autores principales: Muldoon, L. L., Neuwelt, E. A., Pagel, M. A., Weiss, D. L.
Formato: Artigo
Lenguaje:Inglês
Publicado: 1994
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Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC1887364/
https://ncbi.nlm.nih.gov/pubmed/8178934
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