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Year Number of Results
2004 1
2006 1
2007 3
2008 4
2009 2
2010 1
2011 1
2014 3
2015 2
2016 3
2017 7
2018 7
2019 10
2020 7
2021 7
2022 8
2023 11
2024 5

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68 results

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Page 1
Emerging Issues in AAV-Mediated In Vivo Gene Therapy.
Colella P, Ronzitti G, Mingozzi F. Colella P, et al. Among authors: ronzitti g. Mol Ther Methods Clin Dev. 2017 Dec 1;8:87-104. doi: 10.1016/j.omtm.2017.11.007. eCollection 2018 Mar 16. Mol Ther Methods Clin Dev. 2017. PMID: 29326962 Free PMC article. Review.
Semirational bioengineering of AAV vectors with increased potency and specificity for systemic gene therapy of muscle disorders.
El Andari J, Renaud-Gabardos E, Tulalamba W, Weinmann J, Mangin L, Pham QH, Hille S, Bennett A, Attebi E, Bourges E, Leborgne C, Guerchet N, Fakhiri J, Krämer C, Wiedtke E, McKenna R, Guianvarc'h L, Toueille M, Ronzitti G, Hebben M, Mingozzi F, VandenDriessche T, Agbandje-McKenna M, Müller OJ, Chuah MK, Buj-Bello A, Grimm D. El Andari J, et al. Among authors: ronzitti g. Sci Adv. 2022 Sep 23;8(38):eabn4704. doi: 10.1126/sciadv.abn4704. Epub 2022 Sep 21. Sci Adv. 2022. PMID: 36129972 Free PMC article.
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies.
Leborgne C, Barbon E, Alexander JM, Hanby H, Delignat S, Cohen DM, Collaud F, Muraleetharan S, Lupo D, Silverberg J, Huang K, van Wittengerghe L, Marolleau B, Miranda A, Fabiano A, Daventure V, Beck H, Anguela XM, Ronzitti G, Armour SM, Lacroix-Desmazes S, Mingozzi F. Leborgne C, et al. Among authors: ronzitti g. Nat Med. 2020 Jul;26(7):1096-1101. doi: 10.1038/s41591-020-0911-7. Epub 2020 Jun 1. Nat Med. 2020. PMID: 32483358 Free article.
Gene Therapy in Patients with the Crigler-Najjar Syndrome.
D'Antiga L, Beuers U, Ronzitti G, Brunetti-Pierri N, Baumann U, Di Giorgio A, Aronson S, Hubert A, Romano R, Junge N, Bosma P, Bortolussi G, Muro AF, Soumoudronga RF, Veron P, Collaud F, Knuchel-Legendre N, Labrune P, Mingozzi F. D'Antiga L, et al. Among authors: ronzitti g. N Engl J Med. 2023 Aug 17;389(7):620-631. doi: 10.1056/NEJMoa2214084. N Engl J Med. 2023. PMID: 37585628 Clinical Trial.
Evading and overcoming AAV neutralization in gene therapy.
Earley J, Piletska E, Ronzitti G, Piletsky S. Earley J, et al. Among authors: ronzitti g. Trends Biotechnol. 2023 Jun;41(6):836-845. doi: 10.1016/j.tibtech.2022.11.006. Epub 2022 Dec 9. Trends Biotechnol. 2023. PMID: 36503641 Free article. Review.
What's next in gene therapy for Crigler-Najjar syndrome?
Aronson SJ, Ronzitti G, Bosma PJ. Aronson SJ, et al. Among authors: ronzitti g. Expert Opin Biol Ther. 2023 Feb;23(2):119-121. doi: 10.1080/14712598.2022.2160237. Epub 2022 Dec 29. Expert Opin Biol Ther. 2023. PMID: 36579791 No abstract available.
LSD1/PRMT6-targeting gene therapy to attenuate androgen receptor toxic gain-of-function ameliorates spinobulbar muscular atrophy phenotypes in flies and mice.
Prakasam R, Bonadiman A, Andreotti R, Zuccaro E, Dalfovo D, Marchioretti C, Tripathy D, Petris G, Anderson EN, Migazzi A, Tosatto L, Cereseto A, Battaglioli E, Sorarù G, Lim WF, Rinaldi C, Sambataro F, Pourshafie N, Grunseich C, Romanel A, Pandey UB, Contestabile A, Ronzitti G, Basso M, Pennuto M. Prakasam R, et al. Among authors: ronzitti g. Nat Commun. 2023 Feb 6;14(1):603. doi: 10.1038/s41467-023-36186-9. Nat Commun. 2023. PMID: 36746939 Free PMC article.
Gene therapy review: Duchenne muscular dystrophy case study.
Berling E, Nicolle R, Laforêt P, Ronzitti G. Berling E, et al. Among authors: ronzitti g. Rev Neurol (Paris). 2023 Jan-Feb;179(1-2):90-105. doi: 10.1016/j.neurol.2022.11.005. Epub 2022 Dec 12. Rev Neurol (Paris). 2023. PMID: 36517287 Review.
68 results