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Page 1
Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies.
Aguilar-González A, González-Correa JE, Barriocanal-Casado E, Ramos-Hernández I, Lerma-Juárez MA, Greco S, Rodríguez-Sevilla JJ, Molina-Estévez FJ, Montalvo-Romeral V, Ronzitti G, Sánchez-Martín RM, Martín F, Muñoz P. Aguilar-González A, et al. Among authors: ronzitti g. Int J Mol Sci. 2022 Jun 4;23(11):6298. doi: 10.3390/ijms23116298. Int J Mol Sci. 2022. PMID: 35682977 Free PMC article.
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.
Puzzo F, Colella P, Biferi MG, Bali D, Paulk NK, Vidal P, Collaud F, Simon-Sola M, Charles S, Hardet R, Leborgne C, Meliani A, Cohen-Tannoudji M, Astord S, Gjata B, Sellier P, van Wittenberghe L, Vignaud A, Boisgerault F, Barkats M, Laforet P, Kay MA, Koeberl DD, Ronzitti G, Mingozzi F. Puzzo F, et al. Among authors: ronzitti g. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase.
Cagin U, Puzzo F, Gomez MJ, Moya-Nilges M, Sellier P, Abad C, Van Wittenberghe L, Daniele N, Guerchet N, Gjata B, Collaud F, Charles S, Sola MS, Boyer O, Krijnse-Locker J, Ronzitti G, Colella P, Mingozzi F. Cagin U, et al. Among authors: ronzitti g. Mol Ther. 2020 Sep 2;28(9):2056-2072. doi: 10.1016/j.ymthe.2020.05.025. Epub 2020 May 30. Mol Ther. 2020. PMID: 32526204 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.
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.
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
A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III.
Gardin A, Rouillon J, Montalvo-Romeral V, Rossiaud L, Vidal P, Launay R, Vie M, Krimi Benchekroun Y, Cosette J, Bertin B, La Bella T, Dubreuil G, Nozi J, Jauze L, Fragnoud R, Daniele N, Van Wittenberghe L, Esque J, André I, Nissan X, Hoch L, Ronzitti G. Gardin A, et al. Among authors: ronzitti g. J Clin Invest. 2024 Jan 16;134(2):e172018. doi: 10.1172/JCI172018. J Clin Invest. 2024. PMID: 38015640 Free PMC article.
Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.
Jauze L, Vie M, Miagoux Q, Rossiaud L, Vidal P, Montalvo-Romeral V, Saliba H, Jarrige M, Polveche H, Nozi J, Le Brun PR, Bocchialini L, Francois A, Cosette J, Rouillon J, Collaud F, Bordier F, Bertil-Froidevaux E, Georger C, Van Wittenberghe L, Miranda A, Daniele N, Gross D, Hoch L, Nissan X, Ronzitti G. Jauze L, et al. Among authors: ronzitti g. JCI Insight. 2024 May 16:e172614. doi: 10.1172/jci.insight.172614. Online ahead of print. JCI Insight. 2024. PMID: 38753465 Free article.
Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors.
Hösel M, Huber A, Bohlen S, Lucifora J, Ronzitti G, Puzzo F, Boisgerault F, Hacker UT, Kwanten WJ, Klöting N, Blüher M, Gluschko A, Schramm M, Utermöhlen O, Bloch W, Mingozzi F, Krut O, Büning H. Hösel M, et al. Among authors: ronzitti g. Hepatology. 2017 Jul;66(1):252-265. doi: 10.1002/hep.29176. Epub 2017 May 29. Hepatology. 2017. PMID: 28318036 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.
68 results