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多外显子跳跃在犬X连锁肌营养不良用鸡尾酒反义寡核苷酸

JoVE 신문
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JoVE 신문 의학

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy

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多外显子跳跃在犬X连锁肌营养不良用鸡尾酒反义寡核苷酸

DOI:

10.3791/53776-v

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10:30 min

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May 24, 2016

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Bailey Miskew Nichols*¹, Yoshitsugu Aoki*², Mutsuki Kuraoka, Joshua J.A. Lee, Shin’ichi Takeda, Toshifumi Yokota

¹Department of Medical Genetics,University of Alberta Faculty of Medicine and Dentistry, ²Department of Molecular Therapy, National Institute of Neuroscience,National Center of Neurology and Psychiatry

Chapters

00:05Title
01:232’OMePS Transfection of Dog Myoblasts
02:59Morpholino Transfection of Dog Myoblasts and RNA Extraction
05:18Intramuscular Injections or Open Muscle Biopsy
06:42Systemic Injections
07:39Results: Multi-exon Skipping Using Antisense Oligonucleotides in the Dystrophin Gene in Dogs
09:37Conclusion

Summary

자동 번역

外显子跳跃目前是杜氏肌营养不良症（DMD）一个最有希望的治疗选择。扩大对DMD患者的适用性和优化所得截短的肌营养不良蛋白的稳定性/功能，一个多外显子用鸡尾酒的反义寡核苷酸跳过方法被开发，我们证实在狗模型全身肌营养不良蛋白救援。

Tags

Multi-exon Skipping Cocktail Antisense Oligonucleotides Canine X-linked Muscular Dystrophy Duchenne Muscular Dystrophy DMD Antisense Exon Skipping Therapy Dog Model In Vitro Experiments In Vivo Experiments CXMD Myoblasts Cationic Liposome Transfection Differentiation Medium Morpholino Transfection

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