Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

Factor VII (FVII) deficiency is a rare autosomal recessive bleeding disorder treated by infusion of fresh-frozen plasma, plasma-derived FVII concentrates and low-dose recombinant activated FVII. Clinical data suggest that a mild elevation of plasma FVII levels (>10% normal) results in improved he...

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Detalhes bibliográficos
Publicado no:Blood
Main Authors: Marcos-Contreras, Oscar A., Smith, Shannon M., Bellinger, Dwight A., Raymer, Robin A., Merricks, Elizabeth, Faella, Armida, Pavani, Giulia, Zhou, Shangzhen, Nichols, Timothy C., High, Katherine A., Margaritis, Paris
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2016
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4742547/
https://ncbi.nlm.nih.gov/pubmed/26702064
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2015-09-671420
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