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Items: 13


Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice.

Le Quellec S, Dane AP, Barbon E, Bordet JC, Mingozzi F, Dargaud Y, Marais T, Biferi MG, Négrier C, Nathawani AC, Enjolras N.

Thromb Haemost. 2019 Oct 28. doi: 10.1055/s-0039-1697658. [Epub ahead of print]


Gene Therapy for ALS-A Perspective.

Cappella M, Ciotti C, Cohen-Tannoudji M, Biferi MG.

Int J Mol Sci. 2019 Sep 6;20(18). pii: E4388. doi: 10.3390/ijms20184388. Review.


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.

Hum Mol Genet. 2018 Sep 1;27(17):3060-3078. doi: 10.1093/hmg/ddy215.


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.

Sci Transl Med. 2017 Nov 29;9(418). pii: eaam6375. doi: 10.1126/scitranslmed.aam6375.


A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model.

Biferi MG, Cohen-Tannoudji M, Cappelletto A, Giroux B, Roda M, Astord S, Marais T, Bos C, Voit T, Ferry A, Barkats M.

Mol Ther. 2017 Sep 6;25(9):2038-2052. doi: 10.1016/j.ymthe.2017.05.017. Epub 2017 Jun 26.


β-Amyloid Precursor Protein Intracellular Domain Controls Mitochondrial Function by Modulating Phosphatase and Tensin Homolog-Induced Kinase 1 Transcription in Cells and in Alzheimer Mice Models.

Goiran T, Duplan E, Chami M, Bourgeois A, El Manaa W, Rouland L, Dunys J, Lauritzen I, You H, Stambolic V, Biféri MG, Barkats M, Pimplikar SW, Sergeant N, Colin M, Morais VA, Pardossi-Piquard R, Checler F, Alves da Costa C.

Biol Psychiatry. 2018 Mar 1;83(5):416-427. doi: 10.1016/j.biopsych.2017.04.011. Epub 2017 May 3.


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.

Mol Neurodegener. 2016 Jul 28;11(1):58. doi: 10.1186/s13024-016-0123-2.


Intraneuronal aggregation of the β-CTF fragment of APP (C99) induces Aβ-independent lysosomal-autophagic pathology.

Lauritzen I, Pardossi-Piquard R, Bourgeois A, Pagnotta S, Biferi MG, Barkats M, Lacor P, Klein W, Bauer C, Checler F.

Acta Neuropathol. 2016 Aug;132(2):257-276. doi: 10.1007/s00401-016-1577-6. Epub 2016 Apr 30.


Estrogen-mediated downregulation of AIRE influences sexual dimorphism in autoimmune diseases.

Dragin N, Bismuth J, Cizeron-Clairac G, Biferi MG, Berthault C, Serraf A, Nottin R, Klatzmann D, Cumano A, Barkats M, Le Panse R, Berrih-Aknin S.

J Clin Invest. 2016 Apr 1;126(4):1525-37. doi: 10.1172/JCI81894. Epub 2016 Mar 21.


Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice.

Tanguy Y, Biferi MG, Besse A, Astord S, Cohen-Tannoudji M, Marais T, Barkats M.

Front Mol Neurosci. 2015 Jul 28;8:36. doi: 10.3389/fnmol.2015.00036. eCollection 2015.


Proliferation of Multiple Cell Types in the Skeletal Muscle Tissue Elicited by Acute p21 Suppression.

Biferi MG, Nicoletti C, Falcone G, Puggioni EMR, Passaro N, Mazzola A, Pajalunga D, Zaccagnini G, Rizzuto E, Auricchio A, Zentilin L, De Luca G, Giacca M, Martelli F, Musio A, Musarò A, Crescenzi M.

Mol Ther. 2015 May;23(5):885-895. doi: 10.1038/mt.2015.27. Epub 2015 Feb 11.


Cytogenetic analysis of human cells reveals specific patterns of DNA damage in replicative and oncogene-induced senescence.

Falcone G, Mazzola A, Michelini F, Bossi G, Censi F, Biferi MG, Minghetti L, Floridia G, Federico M, Musio A, Crescenzi M.

Aging Cell. 2013 Apr;12(2):312-5. doi: 10.1111/acel.12034. Epub 2012 Dec 25.


Critical requirement for cell cycle inhibitors in sustaining nonproliferative states.

Pajalunga D, Mazzola A, Salzano AM, Biferi MG, De Luca G, Crescenzi M.

J Cell Biol. 2007 Mar 12;176(6):807-18.

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