首页> 外文OA文献 >Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

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Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

机译：morpholino核酸寡聚物与细胞穿透肽共轭的双肌肉生长抑制素和抗肌萎缩蛋白外显子跳跃是一种有前途的治疗策略，用于治疗杜氏肌营养不良症

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Alberto Malerba; Jagjeet K Kang; Graham McClorey; Amer F Saleh; Linda Popplewell; Michael J Gait; Matthew JA Wood; George Dickson;
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年度 2012
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1. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. [J] . Cirak S, Arechavala Gomeza V, Guglieri M, The Lancet . 2011 ,第9791期

机译：系统性二重氨基磷酸吗啉代低聚物治疗后的Duchenne肌营养不良患者的外显子跳跃和肌营养不良蛋白的恢复：一项开放性，2期，剂量递增研究。
2. Immortalized skin fibroblasts expressing conditional MyoD as a renewable and reliable source of converted human muscle cells to assess therapeutic strategies for muscular dystrophies: validation of an exon-skipping approach to restore dystrophin in duchenne muscular dystrophy cells. [J] . Chaouch S, Mouly V, Goyenvalle A, Human gene therapy . 2009 ,第7期

机译：永生化皮肤成纤维细胞将有条件的MyoD表达为转化后的人类肌肉细胞的可再生且可靠的来源，以评估肌肉营养不良的治疗策略：通过外显子跳跃方法恢复杜氏肌营养不良细胞中的肌营养不良蛋白。
3. Dual exon skipping in myostatin and dystrophin for Duchenne muscular dystrophy [J] . Dwi U Kemaladewi, Willem MH Hoogaars, Sandra H van Heiningen, BMC Medical Genomics . 2011 ,第1期

机译：肌生长抑制素和肌营养不良蛋白双重外显子跳跃治疗杜氏肌营养不良症
4. Targeted MS~2 Quantitation of Exon Skipping Restored Dystrophin in a Mouse Model of Duchenne Muscular Dystrophy [C] . Kristy J. Brown, Kitipong Uaesoontrachoon, Aiping Zhang, ASMS Conference on Mass Spectrometry and Allied Topics . 2014

机译：杜南肌营养不良小鼠模型中外显子跳水恢复营养蛋白的靶向MS〜2
5. Experimental and Computational Studies of Structural Differences between Alternative Exon Skipped Repairs for Duchenne Muscular Dystrophy [D] . Ma, Manyuan 2018

机译：实验和计算研究杜兴氏肌营养不良的其他外显子跳过修复之间的结构差异。
6. Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid OligomersConjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for theTreatment of Duchenne Muscular Dystrophy [O] . Alberto Malerba, Jagjeet K Kang, Graham McClorey, 2012

机译：吗啉代核酸低聚物可跳过双重肌肉生长抑制素和肌营养不良蛋白外显子结合细胞穿透肽是一种有前途的治疗策略杜氏肌营养不良症的治疗
7. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: An open-label, phase 2, dose-escalation study [O] . Cirak, S, Arechavala-Gomeza, V, Guglieri, M, 2011

机译：系统性二氨基氨基磷酸吗啉代低聚物治疗后的杜兴氏肌营养不良患者的外显子跳跃和肌营养不良蛋白的恢复：一项开放性，2期，剂量递增研究

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy

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