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Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV techno...
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| Publicado no: | J Inherit Metab Dis |
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| Main Authors: | , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer Netherlands
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5500673/ https://ncbi.nlm.nih.gov/pubmed/28567541 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-017-0053-3 |
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