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Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.
Childers MK, Joubert R, Poulard K, Moal C, Grange RW, Doering JA, Lawlor MW, Rider BE, Jamet T, Danièle N, Martin S, Rivière C, Soker T, Hammer C, Van Wittenberghe L, Lockard M, Guan X, Goddard M, Mitchell E, Barber J, Williams JK, Mack DL, Furth ME, Vignaud A, Masurier C, Mavilio F, Moullier P, Beggs AH, Buj-Bello A. Childers MK, et al. Among authors: daniele n. Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523. Sci Transl Med. 2014. PMID: 24452262 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: daniele n. 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.
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice.
Colella P, Sellier P, Costa Verdera H, Puzzo F, van Wittenberghe L, Guerchet N, Daniele N, Gjata B, Marmier S, Charles S, Simon Sola M, Ragone I, Leborgne C, Collaud F, Mingozzi F. Colella P, et al. Among authors: daniele n. Mol Ther Methods Clin Dev. 2018 Nov 17;12:85-101. doi: 10.1016/j.omtm.2018.11.002. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30581888 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: daniele n. 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: daniele n. 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 NF, Gross DA, Hoch L, Nissan X, Ronzitti G. Jauze L, et al. Among authors: daniele nf. JCI Insight. 2024 May 16;9(11):e172614. doi: 10.1172/jci.insight.172614. JCI Insight. 2024. PMID: 38753465 Free PMC article.
267 results