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Obstacles and future of gene therapy for hemophilia
INTRODUCTION: The recent success of early-phase clinical trials for adeno-associated viral (AAV) liver-directed gene therapy for hemophilia B (HB) demonstrates the potential for gene therapy, in the future, to succeed protein-based prophylaxis therapy for HB. Significant obstacles, however, need to...
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| Publicado en: | Expert Opin Orphan Drugs |
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| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
2015
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4756761/ https://ncbi.nlm.nih.gov/pubmed/26900534 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1517/21678707.2015.1069179 |
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