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Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B
Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for transducing primary human hepatocytes. We have previo...
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| Pubblicato in: | Hum Gene Ther |
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| Autori principali: | , , , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
Mary Ann Liebert, Inc., publishers
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7585622/ https://ncbi.nlm.nih.gov/pubmed/32657150 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.099 |
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