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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Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2016
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Original Article
Länkar: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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