WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration

Wood M.; Zhang J.; Bowman K.; Butler D.; Rinaldi C.; McClorey G.; Frank-Kamenetsky M.; Iwamoto N.; Kothari N.; Lu G.; Mathieu S.; Meena M.; Menon S.; Shimizu M.; Standley S.; Yang H.; Zhong A.; Francis C.; Vargeese C.

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WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration

机译：WVE-210201，对杜南肌营养不良症的调查立体寡核苷酸治疗，诱导外显子51跳跃和染素蛋白恢复

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《Neuromuscular disorders: NMD》 |2017年第2期|共1页
作者
Wood M.; Zhang J.; Bowman K.; Butler D.; Rinaldi C.; McClorey G.; Frank-Kamenetsky M.; Iwamoto N.; Kothari N.; Lu G.; Mathieu S.; Meena M.; Menon S.; Shimizu M.; Standley S.; Yang H.; Zhong A.; Francis C.; Vargeese C.;
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作者单位

Univ Oxford Oxford England;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

Univ Oxford Oxford England;

Univ Oxford Oxford England;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

WAVE Life Sci Ltd Cambridge MA USA;

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原文格式 PDF
正文语种 eng
中图分类神经病学;
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1. WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration [J] . Wood M., Zhang J., Bowman K., Neuromuscular disorders: NMD . 2017,第Suppla2期

机译：WVE-210201，对杜南肌营养不良症的调查立体寡核苷酸治疗，诱导外显子51跳跃和染素蛋白恢复
2. Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping. [J] . Incitti T, De Angelis FG, Cazzella V, Molecular therapy: the journal of the American Society of Gene Therapy . 2010,第9期

机译：外显子跳跃和杜氏肌营养不良症治疗：选择能够诱导肌营养不良蛋白外显子51跳跃的最活跃的U1 snRNA反义。
3. Preclinical studies of WVE-210201, an investigational stereopure antisense oligonucleotide in development for the treatment of patients with duchenne muscular dystrophy (DMD) [J] . M. Panzara, J. Zhang, C. Rinaldi, Journal of the Neurological Sciences: Official Bulletin of the World Federation of Neurology . 2017,第期

机译：WVE-210201的临床前研究，一种杜森恩肌营养不良患者治疗患者的临床研究
4. Dystrophin quantification through high sensitivity mass spectrometry: A novel approach to measure efficacy of exon skipping therapies in Duchenne Muscular Dystrophy [C] . Ken Fantom, Andy West, James Kew American Society for Mass Spectrometry Conference on Mass Spectrometry and Allied Topics . 2012

机译：通过高灵敏度质谱法定量营养不良蛋白定量：一种测量外显子肌营养不良疗效疗效的新方法
5. Combination Therapy Exon Skipping for Duchenne Muscular Dystrophy [D] . Wang, Derek Wu. 2017

机译：跳过杜氏肌营养不良症的联合疗法外显子跳过
6. Exon Skipping and Duchenne Muscular Dystrophy Therapy: Selection of the Most Active U1 snRNA Antisense Able to Induce Dystrophin Exon 51 Skipping [O] . Tania Incitti, Fernanda G De Angelis, Valentina Cazzella, 2010

机译：外显子跳过和杜氏肌营养不良症治疗：最活跃的U1 snRNA反义的选择能够诱导肌营养不良蛋白外显子51跳过。
7. Exon skipping and duchenne muscular dystrophy therapy: selection of the most active U1 snRNA antisense able to induce dystrophin exon 51 skipping. [O] . Tania Incitti, Fernanda G. De Angelis, Valentina Cazzella, 2010

机译：外显子跳跃和杜氏肌营养不良症疗法：选择能够诱导肌营养不良蛋白外显子51跳跃的最活跃的U1 snRNA反义。

WVE-210201, an investigational stereopure oligonucleotide therapy for Duchenne muscular dystrophy, induces Exon 51 skipping and dystrophin protein restoration

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