Sustained correction of bleeding disorder in hemophilia B mice by gene therapy

Mice generated by disrupting the clotting factor IX gene exhibit severe bleeding disorder and closely resemble the phenotype seen in hemophilia B patients. Here we demonstrate that a single intraportal injection of a recombinant adeno-associated virus (AAV) vector encoding canine factor IX cDNA unde...

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Veröffentlicht in:	Proc Natl Acad Sci U S A
Hauptverfasser:	Wang, Lili, Takabe, Kazuaki, Bidlingmaier, Scott M., Ill, Charles R., Verma, Inder M.
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	National Academy of Sciences 1999
Schlagworte:	Biological Sciences
Online-Zugang:	https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC22393/ https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/10097136/ https://ncbi.nlm.nih.govhttps://doi.org/10.1073/pnas.96.7.3906
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