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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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Bibliografski detalji
Izdano u:Hum Gene Ther Clin Dev
Glavni autori: Bradbury, Allison M., Gurda, Brittney L., Casal, Margret L., Ponder, Katherine P., Vite, Charles H., Haskins, Mark E.
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2015
Teme:
Online pristup: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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