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Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease

A preclinical safety study was conducted to evaluate the short- and long-term toxicity of a recombinant adeno-associated virus serotype 8 (AAV2/8) vector that has been developed as an immune-modulatory adjunctive therapy to recombinant human acid α-glucosidase (rhGAA, Myozyme) enzyme replacement tre...

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Detalles Bibliográficos
Autores principales:	Wang, Gensheng, Young, Sarah P, Bali, Deeksha, Hutt, Julie, Li, Songtao, Benson, Janet, Koeberl, Dwight D
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2014
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4362383/ https://www.ncbi.nlm.nih.gov/pubmed/26015962 http://dx.doi.org/10.1038/mtm.2014.18

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