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Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models

BACKGROUND: Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia. However, these pivotal investigations have also identified several challenges. For example, high vector doses are often used...

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Detalles Bibliográficos
Autores principales:	Markusic, David M., Nichols, Timothy C., Merricks, Elizabeth P., Palaschak, Brett, Zolotukhin, Irene, Marsic, Damien, Zolotukhin, Sergei, Srivastava, Arun, Herzog, Roland W.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	BioMed Central 2017
Materias:	Research
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5412045/ https://www.ncbi.nlm.nih.gov/pubmed/28460646 http://dx.doi.org/10.1186/s12967-017-1200-1

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