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Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate. AMT-060 combines an adeno-associated virus-5 (AAV5) vector with a liver-specific promoter driving expr...
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| Publicado no: | Blood |
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| Main Authors: | , , , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Hematology
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5833265/ https://ncbi.nlm.nih.gov/pubmed/29246900 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2017-09-804419 |
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