Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor V...
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
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Milani, Michela , Canepari, Cesare , Liu, Tongyao , Biffi, Mauro , Russo, Fabio , Plati, Tiziana , Curto, Rosalia , Patarroyo-White, Susannah , Drager, Douglas , Visigalli, Ilaria , Brombin, Chiara , Albertini, Paola , Follenzi, Antonia , Ayuso, Eduard , Mueller, Christian , Annoni, Andrea , Naldini, Luigi and Cantore, Alessio
Publisher
London: Nature Publishing Group UK
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English
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Publisher
London: Nature Publishing Group UK
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Contents
Liver gene therapy with adeno-associated viral (AAV) vectors delivering clotting factor transgenes into hepatocytes has shown multiyear therapeutic benefit in adults with hemophilia. However, the mostly episomal nature of AAV vectors challenges their application to young pediatric patients. We developed lentiviral vectors, which integrate in the ho...
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Full title
Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates
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TN_cdi_doaj_primary_oai_doaj_org_article_c9b97a0d651b468fbb352c8d225f07b9
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_c9b97a0d651b468fbb352c8d225f07b9
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-30102-3
