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Long-term correction of hemophilia A mice following lentiviral mediated delivery of an optimized canine factor VIII gene

Current therapies for hemophilia A include frequent prophylactic or on-demand intravenous factor treatments which are costly, inconvenient and may lead to inhibitor formation. Viral vector delivery of factor VIII (FVIII) cDNA has the potential to alleviate the debilitating clotting defects. Lentivir...

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Detalhes bibliográficos
Publicado no:Gene Ther
Main Authors: Staber, JM, Pollpeter, MJ, Anderson, C-G, Burrascano, M, Cooney, AL, Sinn, PL, Rutkowski, DT, Raschke, WC, McCray, PB
Formato: Artigo
Idioma:Inglês
Publicado em: 2017
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5937993/
https://ncbi.nlm.nih.gov/pubmed/28905885
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2017.67
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