Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A.
Esposito F, Lyubenova H, Tornabene P, Auricchio S, Iuliano A, Nusco E, Merlin S, Olgasi C, Manni G, Gargaro M, Fallarino F, Follenzi A, Auricchio A.
Esposito F, et al. Among authors: auricchio a, auricchio s.
EMBO Mol Med. 2022 Jun 8;14(6):e15199. doi: 10.15252/emmm.202115199. Epub 2022 May 2.
EMBO Mol Med. 2022.
PMID: 35491676
Free PMC article.