Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

Enzyme replacement therapy (ERT) is the standard of care for several lysosomal storage diseases (LSDs). ERT, however, requires multiple and costly administrations and has limited efficacy. We recently showed that a single high dose administration of adeno-associated viral vector serotype 8 (AAV2/8)...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5159621/
https://ncbi.nlm.nih.gov/pubmed/27658524
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.181
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