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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: biferi mg. Sci Transl Med. 2017 Nov 29;9(418):eaam6375. doi: 10.1126/scitranslmed.aam6375. Sci Transl Med. 2017. PMID: 29187643 Free PMC article.
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.
Pontoizeau C, Simon-Sola M, Gaborit C, Nguyen V, Rotaru I, Tual N, Colella P, Girard M, Biferi MG, Arnoux JB, Rötig A, Ottolenghi C, de Lonlay P, Mingozzi F, Cavazzana M, Schiff M. Pontoizeau C, et al. Among authors: biferi mg. Nat Commun. 2022 Jun 7;13(1):3278. doi: 10.1038/s41467-022-30880-w. Nat Commun. 2022. PMID: 35672312 Free PMC article.
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: biferi mg. EBioMedicine. 2020 Nov;61:103052. doi: 10.1016/j.ebiom.2020.103052. Epub 2020 Oct 9. EBioMedicine. 2020. PMID: 33039711 Free PMC article.
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. 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.
Lentiviral vector-mediated overexpression of mutant ataxin-7 recapitulates SCA7 pathology and promotes accumulation of the FUS/TLS and MBNL1 RNA-binding proteins.
Alves S, Marais T, Biferi MG, Furling D, Marinello M, El Hachimi K, Cartier N, Ruberg M, Stevanin G, Brice A, Barkats M, Sittler A. Alves S, et al. Among authors: biferi mg. Mol Neurodegener. 2016 Jul 28;11(1):58. doi: 10.1186/s13024-016-0123-2. Mol Neurodegener. 2016. PMID: 27465358 Free PMC article.
26 results