杜氏肌营养不良症与肌营养不良蛋白：发病机制及治疗机遇
Duchenne muscular dystrophy and dystrophin: pathogenesis and opportunities for treatment.
作者信息
Nowak Kristen J, Davies Kay E
机构信息
MRC Functional Genetics Unit, University of Oxford, South Parks Road, Oxford OX1 3QX, UK.
出版信息
EMBO Rep. 2004 Sep;5(9):872-6. doi: 10.1038/sj.embor.7400221.
Abstract
Third in Molecular Medicine Review Series
摘要
分子医学综述系列之三
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本文引用的文献
1
Systemic delivery of genes to striated muscles using adeno-associated viral vectors.使用腺相关病毒载体将基因全身递送至横纹肌。
Nat Med. 2004 Aug;10(8):828-34. doi: 10.1038/nm1085. Epub 2004 Jul 25.
2
LARGE can functionally bypass alpha-dystroglycan glycosylation defects in distinct congenital muscular dystrophies.LARGE可在不同的先天性肌营养不良中通过功能代偿α- dystroglycan糖基化缺陷。
Nat Med. 2004 Jul;10(7):696-703. doi: 10.1038/nm1059. Epub 2004 Jun 6.
3
Muscle-derived stem cells for musculoskeletal tissue regeneration and repair.用于肌肉骨骼组织再生和修复的肌肉衍生干细胞。
Transpl Immunol. 2004 Apr;12(3-4):311-9. doi: 10.1016/j.trim.2003.12.009.
4
Anti-TNFalpha (Remicade) therapy protects dystrophic skeletal muscle from necrosis.抗肿瘤坏死因子α（类克）疗法可保护营养不良性骨骼肌免于坏死。
FASEB J. 2004 Apr;18(6):676-82. doi: 10.1096/fj.03-1024com.
5
Dystrophin expression in myofibers of Duchenne muscular dystrophy patients following intramuscular injections of normal myogenic cells.在杜兴氏肌营养不良症患者的肌纤维中，经肌肉注射正常生肌细胞后的肌营养不良蛋白表达情况。
Mol Ther. 2004 Mar;9(3):475-82. doi: 10.1016/j.ymthe.2003.11.023.
6
Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells.肌肉祖细胞将人微小抗肌萎缩蛋白全身递送至再生的小鼠营养不良性肌肉。
Proc Natl Acad Sci U S A. 2004 Mar 9;101(10):3581-6. doi: 10.1073/pnas.0400373101. Epub 2004 Mar 1.
7
Readthrough of dystrophin stop codon mutations induced by aminoglycosides.氨基糖苷类药物诱导的肌营养不良蛋白终止密码子突变的通读
Ann Neurol. 2004 Mar;55(3):422-6. doi: 10.1002/ana.20052.
8
Long-term expression of full-length human dystrophin in transgenic mdx mice expressing internally deleted human dystrophins.在表达内部缺失型人抗肌萎缩蛋白的转基因mdx小鼠中全长人抗肌萎缩蛋白的长期表达。
Gene Ther. 2004 Jun;11(11):884-93. doi: 10.1038/sj.gt.3302242.
9
Restoration of dystrophin expression in mdx mice by intravascular injection of naked DNA containing full-length dystrophin cDNA.通过血管内注射含全长抗肌萎缩蛋白cDNA的裸DNA来恢复mdx小鼠的抗肌萎缩蛋白表达。
Gene Ther. 2004 Jun;11(11):901-8. doi: 10.1038/sj.gt.3302239.
10
Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector.通过去除辅助依赖性腺病毒载体将全长抗肌萎缩蛋白基因导入mdx小鼠一年后，肌肉功能持续改善。
Hum Gene Ther. 2004 Feb;15(2):145-56. doi: 10.1089/104303404772679959.
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