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Why is AAV FVIII gene therapy not approved by the US Food and Drug Administration yet?
The prospect of a clinical strategy using an adeno-associated virus (AAV) vector for expression of therapeutic levels of factor VIII (FVIII) has been highly desirable. This was initially anticipated by promising data from clinical studies on AAV5-FVIII in men with severe hemophilia A. However, long-...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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American Society of Hematology
2021
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8945649/ https://www.ncbi.nlm.nih.gov/pubmed/34698767 http://dx.doi.org/10.1182/bloodadvances.2021004760 |