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2002 3
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139 results

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Page 1
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.
Safety and efficacy of gene transfer for Leber's congenital amaurosis.
Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J. Maguire AM, et al. Among authors: auricchio a. N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27. N Engl J Med. 2008. PMID: 18441370 Free PMC article. Clinical Trial.
An innovative strategy for the molecular diagnosis of Usher syndrome identifies causal biallelic mutations in 93% of European patients.
Bonnet C, Riahi Z, Chantot-Bastaraud S, Smagghe L, Letexier M, Marcaillou C, Lefèvre GM, Hardelin JP, El-Amraoui A, Singh-Estivalet A, Mohand-Saïd S, Kohl S, Kurtenbach A, Sliesoraityte I, Zobor D, Gherbi S, Testa F, Simonelli F, Banfi S, Fakin A, Glavač D, Jarc-Vidmar M, Zupan A, Battelino S, Martorell Sampol L, Claveria MA, Catala Mora J, Dad S, Møller LB, Rodriguez Jorge J, Hawlina M, Auricchio A, Sahel JA, Marlin S, Zrenner E, Audo I, Petit C. Bonnet C, et al. Among authors: auricchio a. Eur J Hum Genet. 2016 Dec;24(12):1730-1738. doi: 10.1038/ejhg.2016.99. Epub 2016 Jul 27. Eur J Hum Genet. 2016. PMID: 27460420 Free PMC article.
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.
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. Esposito F, et al. Among authors: auricchio a. Cell Rep Med. 2024 Jul 16;5(7):101619. doi: 10.1016/j.xcrm.2024.101619. Epub 2024 Jun 18. Cell Rep Med. 2024. PMID: 38897206 Free PMC article.
Gene Therapy of ABCA4-Associated Diseases.
Auricchio A, Trapani I, Allikmets R. Auricchio A, et al. Cold Spring Harb Perspect Med. 2015 Jan 8;5(5):a017301. doi: 10.1101/cshperspect.a017301. Cold Spring Harb Perspect Med. 2015. PMID: 25573774 Free PMC article. Review.
Liver-directed gene therapy for ornithine aminotransferase deficiency.
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. Boffa I, et al. Among authors: auricchio a. EMBO Mol Med. 2023 Apr 11;15(4):e17033. doi: 10.15252/emmm.202217033. Epub 2023 Jan 17. EMBO Mol Med. 2023. PMID: 36647689 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: 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.
Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.
Ferla R, Dell'Aquila F, Doria M, Ferraiuolo M, Noto A, Grazioli F, Ammendola V, Testa F, Melillo P, Iodice C, Risca G, Tedesco N, le Brun PR, Surace EM, Simonelli F, Galimberti S, Valsecchi MG, Marteau JB, Veron P, Colloca S, Auricchio A. Ferla R, et al. Among authors: auricchio a. Mol Ther Methods Clin Dev. 2023 Feb 11;28:396-411. doi: 10.1016/j.omtm.2023.02.002. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36910588 Free PMC article.
139 results