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

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 well as...

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Detalles Bibliográficos
Autores principales: Esposito, Federica, Lyubenova, Hristiana, Tornabene, Patrizia, Auricchio, Stefano, Iuliano, Antonella, Nusco, Edoardo, Merlin, Simone, Olgasi, Cristina, Manni, Giorgia, Gargaro, Marco, Fallarino, Francesca, Follenzi, Antonia, Auricchio, Alberto
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9174883/
https://www.ncbi.nlm.nih.gov/pubmed/35491676
http://dx.doi.org/10.15252/emmm.202115199

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