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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Detaylı Bibliyografya
Yayımlandı:Mol Ther
Asıl Yazarlar: Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Nature Publishing Group 2016
Konular:
Original Article
Online Erişim: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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