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

A major challenge in the development of a gene therapy for hemophilia A (HA) 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 (LSECs) are the major FVIII source, understand...

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Bibliographic Details

Main Authors:	Rosella Famà, Ester Borroni, Simone Merlin, Chiara Airoldi, Silvia Pignani, Alessia Cucci, Davide Corà, Valentina Bruscaggin, Sharon Scardellato, Stefania Faletti, Giuliana Pelicci, Mirko Pinotti, Gillian E. Walker, Antonia Follenzi
Format:	Artigo
Language:	Inglês
Published:	Ferrata Storti Foundation 2020-05-01
Series:	Haematologica
Online Access:	https://haematologica.org/article/view/9765
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