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Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice.
Kutchukian C, Lo Scrudato M, Tourneur Y, Poulard K, Vignaud A, Berthier C, Allard B, Lawlor MW, Buj-Bello A, Jacquemond V. Kutchukian C, et al. Among authors: vignaud a. Proc Natl Acad Sci U S A. 2016 Dec 13;113(50):14432-14437. doi: 10.1073/pnas.1604099113. Epub 2016 Nov 28. Proc Natl Acad Sci U S A. 2016. PMID: 27911767 Free PMC article.
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: vignaud a. Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523. Sci Transl Med. 2014. PMID: 24452262 Free PMC article.
Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A. Armbruster N, et al. Among authors: vignaud a. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 Free PMC article.
Myocytic androgen receptor controls the strength but not the mass of limb muscles.
Chambon C, Duteil D, Vignaud A, Ferry A, Messaddeq N, Malivindi R, Kato S, Chambon P, Metzger D. Chambon C, et al. Among authors: vignaud a. Proc Natl Acad Sci U S A. 2010 Aug 10;107(32):14327-32. doi: 10.1073/pnas.1009536107. Epub 2010 Jul 26. Proc Natl Acad Sci U S A. 2010. PMID: 20660752 Free PMC article.
DHPR alpha1S subunit controls skeletal muscle mass and morphogenesis.
Piétri-Rouxel F, Gentil C, Vassilopoulos S, Baas D, Mouisel E, Ferry A, Vignaud A, Hourdé C, Marty I, Schaeffer L, Voit T, Garcia L. Piétri-Rouxel F, et al. Among authors: vignaud a. EMBO J. 2010 Feb 3;29(3):643-54. doi: 10.1038/emboj.2009.366. Epub 2009 Dec 24. EMBO J. 2010. PMID: 20033060 Free PMC article.
146 results