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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Detalhes bibliográficos
Publicado no:J Transl Med
Main Authors: Markusic, David M., Nichols, Timothy C., Merricks, Elizabeth P., Palaschak, Brett, Zolotukhin, Irene, Marsic, Damien, Zolotukhin, Sergei, Srivastava, Arun, Herzog, Roland W.
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2017
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5412045/
https://ncbi.nlm.nih.gov/pubmed/28460646
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s12967-017-1200-1
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