Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

We sought to determine whether intramuscular injection of a recombinant adeno-associated virus (rAAV) vector expressing human factor IX (hF.IX) could direct expression of therapeutic levels of the transgene in experimental animals. High titer (10(12)–10(13) vector genomes/ml) rAAV expressing hF.IX w...

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Pubblicato in:	Proc Natl Acad Sci U S A
Autori principali:	Herzog, Roland W., Hagstrom, J. Nathan, Kung, Szu-Hoo, Tai, Shing Jen, Wilson, James M., Fisher, Krishna J., High, Katherine A.
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	National Academy of Sciences 1997
Soggetti:	Biological Sciences
Accesso online:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC20861/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/9159155/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.94.11.5804
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