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In vitro and In vivo Model Systems for Hemophilia A Gene Therapy
Hemophilia A is a hereditary disorder caused by various mutations in factor VIII gene resulting in either a severe deficit or total lack of the corresponding activity. Recent success in gene therapy of a related disease, hemophilia B, gives new hope that similar success can be achieved for hemophili...
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| Veröffentlicht in: | J Genet Syndr Gene Ther |
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| Hauptverfasser: | , , , , , , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2013
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4229687/ https://ncbi.nlm.nih.gov/pubmed/25401041 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4172/2157-7412.S1-014 |
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