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Page 1
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. Sci Transl Med. 2014 Jan 22;6(220):220ra10. doi: 10.1126/scitranslmed.3007523. Sci Transl Med. 2014. PMID: 24452262 Free PMC article.
Muscle function in a canine model of X-linked myotubular myopathy.
Grange RW, Doering J, Mitchell E, Holder MN, Guan X, Goddard M, Tegeler C, Beggs AH, Childers MK. Grange RW, et al. Among authors: childers mk. Muscle Nerve. 2012 Oct;46(4):588-91. doi: 10.1002/mus.23463. Muscle Nerve. 2012. PMID: 22987702 Free PMC article.
Gait characteristics in a canine model of X-linked myotubular myopathy.
Goddard MA, Burlingame E, Beggs AH, Buj-Bello A, Childers MK, Marsh AP, Kelly VE. Goddard MA, et al. Among authors: childers mk. J Neurol Sci. 2014 Nov 15;346(1-2):221-6. doi: 10.1016/j.jns.2014.08.032. Epub 2014 Aug 29. J Neurol Sci. 2014. PMID: 25281397 Free PMC article.
Gene therapy in monogenic congenital myopathies.
Guan X, Goddard MA, Mack DL, Childers MK. Guan X, et al. Among authors: childers mk. Methods. 2016 Apr 15;99:91-8. doi: 10.1016/j.ymeth.2015.10.004. Epub 2015 Oct 14. Methods. 2016. PMID: 26454198 Free PMC article. Review.
Muscle pathology, limb strength, walking gait, respiratory function and neurological impairment establish disease progression in the p.N155K canine model of X-linked myotubular myopathy.
Goddard MA, Mack DL, Czerniecki SM, Kelly VE, Snyder JM, Grange RW, Lawlor MW, Smith BK, Beggs AH, Childers MK. Goddard MA, et al. Among authors: childers mk. Ann Transl Med. 2015 Oct;3(18):262. doi: 10.3978/j.issn.2305-5839.2015.10.31. Ann Transl Med. 2015. PMID: 26605308 Free PMC article.
Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.
Mack DL, Poulard K, Goddard MA, Latournerie V, Snyder JM, Grange RW, Elverman MR, Denard J, Veron P, Buscara L, Le Bec C, Hogrel JY, Brezovec AG, Meng H, Yang L, Liu F, O'Callaghan M, Gopal N, Kelly VE, Smith BK, Strande JL, Mavilio F, Beggs AH, Mingozzi F, Lawlor MW, Buj-Bello A, Childers MK. Mack DL, et al. Among authors: childers mk. Mol Ther. 2017 Apr 5;25(4):839-854. doi: 10.1016/j.ymthe.2017.02.004. Epub 2017 Feb 22. Mol Ther. 2017. PMID: 28237839 Free PMC article.
Long-term effects of systemic gene therapy in a canine model of myotubular myopathy.
Elverman M, Goddard MA, Mack D, Snyder JM, Lawlor MW, Meng H, Beggs AH, Buj-Bello A, Poulard K, Marsh AP, Grange RW, Kelly VE, Childers MK. Elverman M, et al. Among authors: childers mk. Muscle Nerve. 2017 Nov;56(5):943-953. doi: 10.1002/mus.25658. Epub 2017 May 22. Muscle Nerve. 2017. PMID: 28370029 Free PMC article.
87 results