Deciphering the Ets-1/2-mediated transcriptional regulation of F8 gene identifies a minimal F8 promoter for hemophilia A gene therapy

Amajor challenge in the development of a gene therapy for hemophilia A is the selection of cell type- or tissue-specific promoters to ensure factor VIII (FVIII) expression without eliciting an immune response. As liver sinusoidal endothelial cells are the major FVIII source, understanding the transc...

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Detalles Bibliográficos
Autores principales: Famà, Rosella, Borroni, Ester, Merlin, Simone, Airoldi, Chiara, Pignani, Silvia, Cucci, Alessia, Corà, Davide, Bruscaggin, Valentina, Scardellato, Sharon, Faletti, Stefania, Pelicci, Giuliana, Pinotti, Mirko, Walker, Gillian E., Follenzi, Antonia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Fondazione Ferrata Storti 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8168518/
https://www.ncbi.nlm.nih.gov/pubmed/32467137
http://dx.doi.org/10.3324/haematol.2019.239202

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8168518/
https://www.ncbi.nlm.nih.gov/pubmed/32467137
http://dx.doi.org/10.3324/haematol.2019.239202

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