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...

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Detalles Bibliográficos
Autores principales: Barbon, Elena, Kawecki, Charlotte, Marmier, Solenne, Sakkal, Aboud, Collaud, Fanny, Charles, Severine, Ronzitti, Giuseppe, Casari, Caterina, Christophe, Olivier D., Denis, Cécile V., Lenting, Peter J., Mingozzi, Federico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Brief Communication
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

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