Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model

Skeletal muscle–directed expression of FIX-Padua resulted in complete correction of HB phenotype in an inhibitor-prone dog model. Long-term immune tolerance to FIX is sustained over years upon multiple challenges with recombinant FIX protein in 2 HB models.

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Detalhes bibliográficos
Publicado no:Blood Adv
Main Authors: French, Robert A., Samelson-Jones, Benjamin J., Niemeyer, Glenn P., Lothrop, Clinton D., Merricks, Elizabeth P., Nichols, Timothy C., Arruda, Valder R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2018
Assuntos:
Stimulus Report
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5851422/
https://ncbi.nlm.nih.gov/pubmed/29500218
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/bloodadvances.2017015313
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