Ontology highlight
ABSTRACT:
SUBMITTER: Merlin S
PROVIDER: S-EPMC5542771 | biostudies-literature | 2017 Aug
REPOSITORIES: biostudies-literature
Merlin Simone S Cannizzo Elvira Stefania ES Borroni Ester E Bruscaggin Valentina V Schinco Piercarla P Tulalamba Warut W Chuah Marinee K MK Arruda Valder R VR VandenDriessche Thierry T Prat Maria M Valente Guido G Follenzi Antonia A
Molecular therapy : the journal of the American Society of Gene Therapy 20170526 8
Hemophilia A (HA) is an X-linked bleeding disease caused by factor VIII (FVIII) deficiency. We previously demonstrated that FVIII is produced specifically in liver sinusoid endothelial cells (LSECs) and to some degree in myeloid cells, and thus, in the present work, we seek to restrict the expression of FVIII transgene to these cells using cell-specific promoters. With this approach, we aim to limit immune response in a mouse model by lentiviral vector (LV)-mediated gene therapy encoding FVIII. ...[more]