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Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor
Von Willebrand disease (VWD), the most common inherited bleeding disorder in humans, is caused by quantitative or qualitative defects in von Willebrand factor (VWF). VWD represents a potential target for gene therapy applications, as a single treatment could potentially result in a long-term correct...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10113149/ https://www.ncbi.nlm.nih.gov/pubmed/33456057 http://dx.doi.org/10.1038/s41434-020-00218-6 |