1. Dudley RW, Lu Y, Gilbert R, Matecki S, Nalbantoglu J, Petrof BJ, Karpati G. Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector. Hum Gene Ther. 2004 Feb;15(2):145-56. doi: 10.10...
Dmdmdx mice have defective oligodendrogenesis, delayed myelin compaction and persistent hypomyelinationDystrophinDystrophin-glycoprotein complexDuchenne muscular dystrophyOligodendrocyteOligodendrocyte progenitor cellMyelinationMyelin compactionCorpus callosumDuchenne muscular dystrophy (DMD) is caused by mutations in the...
14.Wang M, Tucker JD, Lu P, Wu B, Cloer C, Lu Q. Tris[2-(acryloyloxy)ethyl]isocyanurate cross-linked low-molecular-weight polyethylenimine as gene delivery carriers in cell culture and dystrophic mdx mice. Bioconjug Chem. 2012; 23:837–845. 15.Kobinger GP, Louboutin JP, Barton ER, Sw...
[1] Ryder-Cook AS, Sicinski P, Thomas K, et al. Localization of the mdx mutation within the mouse dystrophin gene. [J]. EMBO,1988.[2] Mizobe Y , Miyatake S , Takizawa H , et al. In Vivo Evaluation of Single-Exon and Multiexon Skipping in mdx52 Mice[J].Methods Mol Biol, 2018...
[2]Hermes TA, Kido LA, Macedo AB, Mizobuti DS, Moraes LHR, Somazz MC, Cagnon VHA, Minatel E. Sex influences diaphragm muscle response in exercised mdx mice. Cell Biol Int. 2018 Dec;42(12):1611-1621. [3]Salimena MC, Lagrota-Candido J, Quírico-Santos T. Gender dimorphism influence...
DMD基因是人体已知最大的蛋白编码基因,长度约2.4 Mb。超过75%的患者存在DMD基因的大片段缺失或无义突变,且突变集中于第3-9号和第45-55号外显子区域。这些突变导致肌营养不良蛋白缺失,并进一步引发肌营养不良蛋白相关糖蛋白复合物(DGC)的分解,破坏肌动蛋白与细胞外基质间的相互作用,使肌肉极易受损。这种肌肉易损...
delivery of rAAVrh74. MHCK7. micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial[J]. JAMA neurology, 2020, 77(9): 1122-1131.Wells D J, Wells K E, Asante E A, et al. Expression of human full-length and minidystrophin in transgenic mdx mice:...
而mdx小鼠非常不适合用于ASO跳跃疗法,特别是ASO药物特异性跳跃23号之后的外显子的情况下,因为mdx小鼠在E23上已经有终止子,而跳跃其后的外显子无法带来行为学方面的药效反应,仅通过mRNA表达水平来评估药效是匮乏的。 有的文献还使用了hDMDΔ52/mdx模型检测ASO跳跃疗法的药效[3],该模型是通过TG转入人全长DMD并敲除E...
Wells D J, Wells K E, Asante E A, et al. Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy[J]. Human Molecular Genetics, 1995, 4(8): 1245-1250. ...
22. Hourdé, C.et al.Voluntary Physical Activity Protects from Susceptibility to Skeletal Muscle Contraction–Induced Injury But Worsens Heart Function in mdx Mice.Am. J. Pathol.182, 1509–1518 (2013). 23. Morroni, J.et al.Accelerating the Mdx Heart Histo-Pathology through Physical Exercise.Li...