Pubblicazioni

Pubblicazioni InnovAAVector dal 2021 a oggi

  1. P. Tornabene, I. Trapani, M. Centrulo, E. Marrocco, R. Minopoli, M. Lupo, C. Iodice, C. Gesualdo, F. Simonelli, E.M. Surace, A. Auricchio “Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina” Molecular Therapy – Methods & Clinica Development, 2021
  2. 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. Therapeutic homology-independent targeted integration in retina and liver. Nat Commun. 2022 Apr 12;13(1):1963. doi: 10.1038/s41467-022-29550-8. PMID: 35414130; PMCID: PMC9005519.
  3. 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. Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A. EMBO Mol Med. 2022 Jun 8;14(6):e15199. doi: 10.15252/emmm.202115199. Epub 2022 May 2. PMID: 35491676; PMCID: PMC9174883.
  4. Di Lorenzo G, Iavarone F, Maddaluno M, Plata-Gómez AB, Aureli S, Quezada Meza CP, Cinque L, Palma A, Reggio A, Cirillo C, Sacco F, Stolz A, Napolitano G, Marin O, Pinna LA, Ruzzene M, Limongelli V, Efeyan A, Grumati P, Settembre C. Phosphorylation of FAM134C by CK2 controls starvation-induced ER-phagy. Sci Adv. 2022 Sep 2;8(35):eabo1215. doi: 10.1126/sciadv.abo1215. Epub 2022 Aug 31. PMID: 36044577; PMCID: PMC9432840.
  5. Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, Calabria A, Vavassori V, Cipriani C, Castiello MC, Esposito S, Brombin C, Cugnata F, Adjali O, Ayuso E, Merelli I, Villa A, Di Micco R, Kajaste-Rudnitski A, Montini E, Penaud-Budloo M, Naldini L. Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells. Cell Stem Cell. 2022 Oct 6;29(10):1428-1444.e9. doi: 10.1016/j.stem.2022.09.001. PMID: 36206730.
  6. Boffa I, Polishchuk E, De Stefano L, Dell’Aquila F, Nusco E, Marrocco E, Audano M, Pedretti S, Caterino M, Bellezza I, Ruoppolo M, Mitro N, Cellini B, Auricchio A, Brunetti-Pierri N. Liver-directed gene therapy for ornithine aminotransferase deficiency. EMBO Mol Med. 2023 Apr 11;15(4):e17033. doi: 10.15252/emmm.202217033. Epub 2023 Jan 17. PMID: 36647689; PMCID: PMC10086579.
  7. Padula A, Spinelli M, Nusco E, Bujanda Cundin X, Capolongo F, Campione S, Perna C, Bastille A, Ericson M, Wang CC, Zhang S, Amoresano A, Nacht M, Piccolo P. Genome editing without nucleases confers proliferative advantage to edited hepatocytes and corrects Wilson disease. JCI Insight. 2023 Nov 8;8(21):e171281. doi: 10.1172/jci.insight.171281. PMID: 37707949; PMCID: PMC10721260.
  8. Pedrazzoli, E., Demozzi, M., Visentin, E. et al. CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing. Nat Commun 15, 3478 (2024). https://doi.org/10.1038/s41467-024-47800-9
  9. Esposito F, Dell’Aquila F, Rhiel M, Auricchio S, Chmielewski KO, Andrieux G, Ferla R, Horrach PS, Padmanabhan A, Di Cunto R, Notaro S, Santeularia ML, Boerries M, Dell’Anno M, Nusco E, Padula A, Nutarelli S, Cornu TI, Sorrentino NC, Piccolo P, Trapani I, Cathomen T, Auricchio A. Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases. Cell Rep Med. 2024 Jul 16;5(7):101619. doi: 10.1016/j.xcrm.2024.101619. Epub 2024 Jun 18. PMID: 38897206; PMCID: PMC11293346.
  10. Bernardo-Colón A, Bighinati A, Parween S, Debnath S, Piano I, Adani E, Corsi F, Gargini C, Vergara N, Marigo V, Becerra SP. H105A peptide eye drops promote photoreceptor survival in murine and human models of retinal degeneration. bioRxiv [Preprint]. 2024 Jul 16:2024.07.10.602890. doi: 10.1101/2024.07.10.602890. Update in: Commun Med (Lond). 2025 Mar 21;5(1):81. doi: 10.1038/s43856-025-00789-8. PMID: 39109177; PMCID: PMC11302621.
  11. Giaccio M, Monaco A, Galiano L, Parente A, Borzacchiello L, Rubino R, Klärner FG, Killa D, Perna C, Piccolo P, Marotta M, Pan X, Khijniak M, Siddique I, Schrader T, Pshezhetsky AV, Sorrentino NC, Bitan G, Fraldi A. Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA. Mol Ther. 2024 Sep 28:S1525-0016(24)00654-3. doi: 10.1016/j.ymthe.2024.09.030. Epub ahead of print. PMID: 39342429.
  12. Ferla R, Pugni E, Lupo M, Tiberi P, Fioretto F, Perota A, Duchi R, Lagutina I, Gesualdo C, Rossi S, Ventrella D, Elmi A, McClinton B, Toomes C, Xu T, Molday RS, Surace EM, Simonelli F, Bacci ML, Galli C, Memon MA, Shams N, Auricchio A, Trapani I. Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models. Sci Adv. 2025 Mar 28;11(13):eadt9354. doi: 10.1126/sciadv.adt9354. Epub 2025 Mar 26. PMID: 40138422; PMCID: PMC11939046.
  13. Ferriero R, Bruno G, Padula A, Pisano S, Boffa I, Gargaro M, Imperatore T, Battipaglia M, Vivenzio S, Perna C, Nusco E, Ferrante L, Westhaus A, Knight M, Manni G, Campione S, Di Napoli E, Polishchuk E, Polishchuk R, Paciello O, Brunetti-Pierri N, Lisowski L, Fallarino F, Piccolo P. Impact of liver fibrosis on AAV-mediated gene transfer to mouse hepatocytes. Nat Commun. 2025 Mar 10;16(1):2118. doi: 10.1038/s41467-025-57382-9. PMID: 40064861; PMCID: PMC11893804.
  14. Dell’Aquila F, Di Cunto R, Marrocco E, Del Prete E, D’Alessio A, De Stefano L, Notaro S, Nusco E, Auricchio A. Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina. Mol Ther. 2025 Jul 2;33(7):2997-3005. doi: 10.1016/j.ymthe.2024.10.019. Epub 2024 Oct 28. PMID: 39489914; PMCID: PMC12265955.
  15. Pisani M, Calandra F, Rinaldi A, Cella F, Tedeschi F, Boffa I, Vozzi D, Brunetti-Pierri N, Carissimo A, Napolitano F, Siciliano V. Computational identification of small molecules for increased gene expression by synthetic circuits in mammalian cells. Nat Commun. 2025 Aug 4;16(1):7160. doi: 10.1038/s41467-025-62529-9. PMID: 40759644; PMCID: PMC12322086.
  16. Donati V, Di Pietro C, Persano L, Rampazzo E, Panarelli M, Cambria C, Selimi A, Manfreda L, de Oliveira do Rêgo AG, La Sala G, Sprega C, Calistri A, Ciubotaru CD, Yang G, Zonta F, Antonucci F, Marazziti D, Mammano F. Connexin hemichannel blockade by abEC1.1 disrupts glioblastoma progression, suppresses invasiveness, and reduces hyperexcitability in preclinical models. Cell Commun Signal. 2025 Sep 2;23(1):391. doi: 10.1186/s12964-025-02370-1. PMID: 40898342; PMCID: PMC12403430.
  17. Medici G, Tassinari M, Loi M, Bove AM, Garofani BC, Volpedo G, Mottolese N, Matteoli G, Lo Martire V, Berteotti C, Candini G, Trebbi F, Riva A, Striano P, Zoccoli G, Curia G, Trazzi S, Ciani E. Safety and efficacy of Igk-TATk-CDKL5 gene therapy in mosaic CDKL5 deficiency. Neurotherapeutics. 2025 Oct;22(6):e00727. doi: 10.1016/j.neurot.2025.e00727. Epub 2025 Sep 2. PMID: 40903342; PMCID: PMC12664459.

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