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A Review of Gene Therapy in Canine and Feline Models of Lysosomal Storage Disorders

Lysosomal storage disorders (LSDs) are inherited diseases that result from the intracellular accumulation of incompletely degraded macromolecules. The majority of LSDs affect both the peripheral and central nervous systems and are not effectively treated by enzyme replacement therapy, substrate redu...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther Clin Dev
Main Authors: Bradbury, Allison M., Gurda, Brittney L., Casal, Margret L., Ponder, Katherine P., Vite, Charles H., Haskins, Mark E.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4516914/
https://ncbi.nlm.nih.gov/pubmed/25671613
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2015.002
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