AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune response to vector capsid is clearly dose dependent. To decrease the vector dose, we explored AAV-8 (1-3 × 10(12) vg/kg) encoding a hyperfunctiona...

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Detalhes bibliográficos
Publicado no:Blood
Main Authors: Crudele, Julie M., Finn, Jonathan D., Siner, Joshua I., Martin, Nicholas B., Niemeyer, Glenn P., Zhou, Shangzhen, Mingozzi, Federico, Lothrop, Clinton D., Arruda, Valder R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2015
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4351503/
https://ncbi.nlm.nih.gov/pubmed/25568350
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2014-07-588194
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