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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.
Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome.
Parrini M, Naskar S, Alberti M, Colombi I, Morelli G, Rocchi A, Nanni M, Piccardi F, Charles S, Ronzitti G, Mingozzi F, Contestabile A, Cancedda L. Parrini M, et al. Among authors: ronzitti g. Mol Ther. 2021 Oct 6;29(10):3072-3092. doi: 10.1016/j.ymthe.2021.05.023. Epub 2021 May 29. Mol Ther. 2021. PMID: 34058387 Free PMC article.
Parkin regulates kainate receptors by interacting with the GluK2 subunit.
Maraschi A, Ciammola A, Folci A, Sassone F, Ronzitti G, Cappelletti G, Silani V, Sato S, Hattori N, Mazzanti M, Chieregatti E, Mulle C, Passafaro M, Sassone J. Maraschi A, et al. Among authors: ronzitti g. Nat Commun. 2014 Oct 15;5:5182. doi: 10.1038/ncomms6182. Nat Commun. 2014. PMID: 25316086 Free PMC article.
Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.
Meliani A, Boisgerault F, Hardet R, Marmier S, Collaud F, Ronzitti G, Leborgne C, Costa Verdera H, Simon Sola M, Charles S, Vignaud A, van Wittenberghe L, Manni G, Christophe O, Fallarino F, Roy C, Michaud A, Ilyinskii P, Kishimoto TK, Mingozzi F. Meliani A, et al. Among authors: ronzitti g. Nat Commun. 2018 Oct 5;9(1):4098. doi: 10.1038/s41467-018-06621-3. Nat Commun. 2018. PMID: 30291246 Free PMC article.
SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson's Disease.
Espinoza S, Scarpato M, Damiani D, Managò F, Mereu M, Contestabile A, Peruzzo O, Carninci P, Santoro C, Papaleo F, Mingozzi F, Ronzitti G, Zucchelli S, Gustincich S. Espinoza S, et al. Among authors: ronzitti g. Mol Ther. 2020 Feb 5;28(2):642-652. doi: 10.1016/j.ymthe.2019.08.005. Epub 2019 Aug 16. Mol Ther. 2020. PMID: 31495777 Free PMC article.
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.
Costa-Verdera H, Collaud F, Riling CR, Sellier P, Nordin JML, Preston GM, Cagin U, Fabregue J, Barral S, Moya-Nilges M, Krijnse-Locker J, van Wittenberghe L, Daniele N, Gjata B, Cosette J, Abad C, Simon-Sola M, Charles S, Li M, Crosariol M, Antrilli T, Quinn WJ 3rd, Gross DA, Boyer O, Anguela XM, Armour SM, Colella P, Ronzitti G, Mingozzi F. Costa-Verdera H, et al. Among authors: ronzitti g. Nat Commun. 2021 Nov 4;12(1):6393. doi: 10.1038/s41467-021-26744-4. Nat Commun. 2021. PMID: 34737297 Free PMC article.
Combination Therapy Is the New Gene Therapy?
Ronzitti G, Mingozzi F. Ronzitti G, et al. Mol Ther. 2018 Jan 3;26(1):12-14. doi: 10.1016/j.ymthe.2017.12.008. Epub 2017 Dec 20. Mol Ther. 2018. PMID: 29273499 Free PMC article. No abstract available.
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.
Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa-/- mice.
Sellier P, Vidal P, Bertin B, Gicquel E, Bertil-Froidevaux E, Georger C, van Wittenberghe L, Miranda A, Daniele N, Richard I, Gross DA, Mingozzi F, Collaud F, Ronzitti G. Sellier P, et al. Among authors: ronzitti g. J Inherit Metab Dis. 2024 Jan;47(1):119-134. doi: 10.1002/jimd.12625. Epub 2023 May 29. J Inherit Metab Dis. 2024. PMID: 37204237
67 results