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

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 devel...

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Detalles Bibliográficos
Autores principales: 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, Cantore, Alessio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9068791/
https://www.ncbi.nlm.nih.gov/pubmed/35508619
http://dx.doi.org/10.1038/s41467-022-30102-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9068791/
https://www.ncbi.nlm.nih.gov/pubmed/35508619
http://dx.doi.org/10.1038/s41467-022-30102-3

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