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Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

Authors :
Michela Milani
Cesare Canepari
Tongyao Liu
Mauro Biffi
Fabio Russo
Tiziana Plati
Rosalia Curto
Susannah Patarroyo-White
Douglas Drager
Ilaria Visigalli
Chiara Brombin
Paola Albertini
Antonia Follenzi
Eduard Ayuso
Christian Mueller
Andrea Annoni
Luigi Naldini
Alessio Cantore
Source :
Nature Communications, Vol 13, Iss 1, Pp 1-14 (2022)
Publication Year :
2022
Publisher :
Nature Portfolio, 2022.

Abstract

“Lentiviral gene therapy to the liver establishes stable long-term normal to supra-normal coagulation factor VIII activity in mouse models of hemophilia A and in non-human primates, representing a potential new treatment option for people with hemophilia A.”.

Subjects

Subjects :
Science

Details

Language :
English
ISSN :
20411723
Volume :
13
Issue :
1
Database :
Directory of Open Access Journals
Journal :
Nature Communications
Publication Type :
Academic Journal
Accession number :
edsdoj.9b97a0d651b468fbb352c8d225f07b9
Document Type :
article
Full Text :
https://doi.org/10.1038/s41467-022-30102-3