Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A

Abstract Liver gene therapy with adeno‐associated viral (AAV) vectors is under clinical investigation for haemophilia A (HemA), the most common inherited X‐linked bleeding disorder. Major limitations are the large size of the F8 transgene, which makes packaging in a single AAV vector a challenge, as...

Full description

Bibliographic Details
Main Authors: Federica Esposito, Hristiana Lyubenova, Patrizia Tornabene, Stefano Auricchio, Antonella Iuliano, Edoardo Nusco, Simone Merlin, Cristina Olgasi, Giorgia Manni, Marco Gargaro, Francesca Fallarino, Antonia Follenzi, Alberto Auricchio
Format: Article
Language:English
Published: Springer Nature 2022-06-01
Series:EMBO Molecular Medicine
Subjects:
AAV vectors
haemophilia A
liver gene therapy
protein trans‐splicing
Online Access:https://doi.org/10.15252/emmm.202115199

Internet

https://doi.org/10.15252/emmm.202115199

Similar Items