Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

Preclinical studies and initial clinical trials have documented the feasibility of adenoassociated virus (AAV)–mediated gene therapy for hemophilia B. In an 8-year study, inhibitor-prone hemophilia B dogs (n = 2) treated with liver-directed AAV2 factor IX (FIX) gene therapy did not have a single ble...

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Библиографические подробности
Главные авторы: Niemeyer, Glenn P., Herzog, Roland W., Mount, Jane, Arruda, Valder R., Tillson, D. Michael, Hathcock, John, van Ginkel, Frederik W., High, Katherine A., Lothrop, Clinton D.
Формат: Artigo
Язык:Inglês
Опубликовано: American Society of Hematology 2009
Предметы:
Gene Therapy
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC2630266/
https://ncbi.nlm.nih.gov/pubmed/18957684
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2008-10-181479
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