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2. Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice. Danièle N; Moal C; Julien L; Marinello M; Jamet T; Martin S; Vignaud A; Lawlor MW; Buj-Bello A J Neuropathol Exp Neurol; 2018 Apr; 77(4):282-295. PubMed ID: 29408998 [TBL] [Abstract][Full Text] [Related]
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4. Cellular, biochemical and molecular changes in muscles from patients with X-linked myotubular myopathy due to MTM1 mutations. Bachmann C; Jungbluth H; Muntoni F; Manzur AY; Zorzato F; Treves S Hum Mol Genet; 2017 Jan; 26(2):320-332. PubMed ID: 28007904 [TBL] [Abstract][Full Text] [Related]
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7. Site-specific Mtm1 mutagenesis by an AAV-Cre vector reveals that myotubularin is essential in adult muscle. Joubert R; Vignaud A; Le M; Moal C; Messaddeq N; Buj-Bello A Hum Mol Genet; 2013 May; 22(9):1856-66. PubMed ID: 23390130 [TBL] [Abstract][Full Text] [Related]
16. rAAV-related therapy fully rescues myonuclear and myofilament function in X-linked myotubular myopathy. Ross JA; Tasfaout H; Levy Y; Morgan J; Cowling BS; Laporte J; Zanoteli E; Romero NB; Lowe DA; Jungbluth H; Lawlor MW; Mack DL; Ochala J Acta Neuropathol Commun; 2020 Oct; 8(1):167. PubMed ID: 33076971 [TBL] [Abstract][Full Text] [Related]
17. Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C mice. Lawlor MW; Viola MG; Meng H; Edelstein RV; Liu F; Yan K; Luna EJ; Lerch-Gaggl A; Hoffmann RG; Pierson CR; Buj-Bello A; Lachey JL; Pearsall S; Yang L; Hillard CJ; Beggs AH Am J Pathol; 2014 Jun; 184(6):1831-42. PubMed ID: 24726641 [TBL] [Abstract][Full Text] [Related]
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20. X-linked myotubular myopathy in a family with two infant siblings: a case with MTM1 mutation. Jeon JH; Namgung R; Park MS; Park KI; Lee C; Lee JS; Kim SH Yonsei Med J; 2011 May; 52(3):547-50. PubMed ID: 21488203 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]