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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...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Egile Nagusiak: Wang, Lili, Takabe, Kazuaki, Bidlingmaier, Scott M., Ill, Charles R., Verma, Inder M.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: The National Academy of Sciences 1999
Gaiak:
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC22393/
https://ncbi.nlm.nih.gov/pubmed/10097136
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