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Therapeutic homology-independent targeted integration in retina and liver.
Tornabene P, Ferla R, Llado-Santaeularia M, Centrulo M, Dell'Anno M, Esposito F, Marrocco E, Pone E, Minopoli R, Iodice C, Nusco E, Rossi S, Lyubenova H, Manfredi A, Di Filippo L, Iuliano A, Torella A, Piluso G, Musacchia F, Surace EM, Cacchiarelli D, Nigro V, Auricchio A. Tornabene P, et al. Among authors: auricchio a. Nat Commun. 2022 Apr 12;13(1):1963. doi: 10.1038/s41467-022-29550-8. Nat Commun. 2022. PMID: 35414130 Free PMC article.
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.
Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina.
Tornabene P, Trapani I, Minopoli R, Centrulo M, Lupo M, de Simone S, Tiberi P, Dell'Aquila F, Marrocco E, Iodice C, Iuliano A, Gesualdo C, Rossi S, Giaquinto L, Albert S, Hoyng CB, Polishchuk E, Cremers FPM, Surace EM, Simonelli F, De Matteis MA, Polishchuk R, Auricchio A. Tornabene P, et al. Among authors: auricchio a. Sci Transl Med. 2019 May 15;11(492):eaav4523. doi: 10.1126/scitranslmed.aav4523. Sci Transl Med. 2019. PMID: 31092694 Free PMC article.
Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteintrans-splicing in the retina.
Tornabene P, Trapani I, Centrulo M, Marrocco E, Minopoli R, Lupo M, Iodice C, Gesualdo C, Simonelli F, Surace EM, Auricchio A. Tornabene P, et al. Among authors: auricchio a. Mol Ther Methods Clin Dev. 2021 Oct 19;23:448-459. doi: 10.1016/j.omtm.2021.10.004. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34786437 Free PMC article.
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Padula A, Petruzzelli R, Philbert SA, Church SJ, Esposito F, Campione S, Monti M, Capolongo F, Perna C, Nusco E, Schmidt HH, Auricchio A, Cooper GJS, Polishchuk R, Piccolo P. Padula A, et al. Among authors: auricchio a. Mol Ther Methods Clin Dev. 2022 Aug 13;26:495-504. doi: 10.1016/j.omtm.2022.08.004. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092366 Free PMC article.
Versatility of AAV vectors for retinal gene transfer.
Surace EM, Auricchio A. Surace EM, et al. Among authors: auricchio a. Vision Res. 2008 Feb;48(3):353-9. doi: 10.1016/j.visres.2007.07.027. Epub 2007 Oct 17. Vision Res. 2008. PMID: 17923143 Free article. Review.
708 results