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Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy
Hemophilia B, a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), is an excellent candidate for gene therapy. However, to date, success in hemophilia gene therapy clinical trials has been limited due to failure to achieve or sustain therapeutic levels of factor expr...
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| Publicado no: | Gene Ther |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2011
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6070132/ https://ncbi.nlm.nih.gov/pubmed/21412285 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2011.31 |
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