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Triple Vectors Expand AAV Transfer Capacity in the Retina.
Maddalena A, Tornabene P, Tiberi P, Minopoli R, Manfredi A, Mutarelli M, Rossi S, Simonelli F, Naggert JK, Cacchiarelli D, Auricchio A. Maddalena A, et al. Among authors: minopoli r. Mol Ther. 2018 Feb 7;26(2):524-541. doi: 10.1016/j.ymthe.2017.11.019. Epub 2017 Dec 5. Mol Ther. 2018. PMID: 29292161 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: minopoli r. Sci Transl Med. 2019 May 15;11(492):eaav4523. doi: 10.1126/scitranslmed.aav4523. Sci Transl Med. 2019. PMID: 31092694 Free PMC article.
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: minopoli r. Nat Commun. 2022 Apr 12;13(1):1963. doi: 10.1038/s41467-022-29550-8. Nat Commun. 2022. PMID: 35414130 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: minopoli r. 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.