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Gene therapy for hemophilia: what does the future hold?
Recent phase I/II adeno-associated viral vector-mediated gene therapy clinical trials have reported remarkable success in ameliorating disease phenotype in hemophilia A and B. These trials, which highlight the challenges overcome through decades of preclinical and first in human clinical studies, ha...
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| Veröffentlicht in: | Ther Adv Hematol |
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| Hauptverfasser: | , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
SAGE Publications
2018
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6130099/ https://ncbi.nlm.nih.gov/pubmed/30210756 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/2040620718791933 |
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