Nature Communications (May 2022)
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
- 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
Affiliations
- Michela Milani
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Cesare Canepari
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Tongyao Liu
- Sanofi
- Mauro Biffi
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Fabio Russo
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Tiziana Plati
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Rosalia Curto
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Susannah Patarroyo-White
- Sanofi
- Douglas Drager
- Sanofi
- Ilaria Visigalli
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Chiara Brombin
- University Center for Statistics in the Biomedical Sciences, Vita-Salute San Raffaele University
- Paola Albertini
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Antonia Follenzi
- Department of Health Sciences, University of Piemonte Orientale
- Eduard Ayuso
- INSERM UMR1089, University of Nantes, CHU de Nantes
- Christian Mueller
- Sanofi
- Andrea Annoni
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Luigi Naldini
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- Alessio Cantore
- San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute
- DOI
- https://doi.org/10.1038/s41467-022-30102-3
- Journal volume & issue
-
Vol. 13,
no. 1
pp. 1 – 14
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.”.
