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Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.
Colella P, Sellier P, Gomez MJ, Biferi MG, Tanniou G, Guerchet N, Cohen-Tannoudji M, Moya-Nilges M, van Wittenberghe L, Daniele N, Gjata B, Krijnse-Locker J, Collaud F, Simon-Sola M, Charles S, Cagin U, Mingozzi F. Colella P, et al. Among authors: cohen tannoudji m. EBioMedicine. 2020 Nov;61:103052. doi: 10.1016/j.ebiom.2020.103052. Epub 2020 Oct 9. EBioMedicine. 2020. PMID: 33039711 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. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Gene Therapy for ALS-A Perspective.
Cappella M, Ciotti C, Cohen-Tannoudji M, Biferi MG. Cappella M, et al. Int J Mol Sci. 2019 Sep 6;20(18):4388. doi: 10.3390/ijms20184388. Int J Mol Sci. 2019. PMID: 31500113 Free PMC article. Review.
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.
Biferi MG, Cohen-Tannoudji M, García-Silva A, Souto-Rodríguez O, Viéitez-González I, San-Millán-Tejado B, Fernández-Carrera A, Pérez-Márquez T, Teijeira-Bautista S, Barrera S, Domínguez V, Marais T, González-Fernández Á, Barkats M, Ortolano S. Biferi MG, et al. Among authors: cohen tannoudji m. Mol Ther Methods Clin Dev. 2020 Oct 22;20:1-17. doi: 10.1016/j.omtm.2020.10.016. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33335943 Free PMC article.
Cofilin-1 phosphorylation catalyzed by ERK1/2 alters cardiac actin dynamics in dilated cardiomyopathy caused by lamin A/C gene mutation.
Chatzifrangkeskou M, Yadin D, Marais T, Chardonnet S, Cohen-Tannoudji M, Mougenot N, Schmitt A, Crasto S, Di Pasquale E, Macquart C, Tanguy Y, Jebeniani I, Pucéat M, Morales Rodriguez B, Goldmann WH, Dal Ferro M, Biferi MG, Knaus P, Bonne G, Worman HJ, Muchir A. Chatzifrangkeskou M, et al. Hum Mol Genet. 2018 Sep 1;27(17):3060-3078. doi: 10.1093/hmg/ddy215. Hum Mol Genet. 2018. PMID: 29878125 Free PMC article.
81 results