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Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children
Recombinant AAV serotype vectors and their variants have been or are currently being used for gene therapy for hemophilia in several phase I/II/III clinical trials in humans. However, none of these trials have included children with hemophilia since the traditional liver-directed AAV gene therapy wi...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Frontiers Media S.A.
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9583144/ https://www.ncbi.nlm.nih.gov/pubmed/36274690 http://dx.doi.org/10.3389/fmicb.2022.1033615 |