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DOI :
10.3791/53776-v
•
10:30 min
May 24th, 2016
Chapters
0:05
Title
1:23
2'OMePS Transfection of Dog Myoblasts
2:59
Morpholino Transfection of Dog Myoblasts and RNA Extraction
5:18
Intramuscular Injections or Open Muscle Biopsy
6:42
Systemic Injections
7:39
Results: Multi-exon Skipping Using Antisense Oligonucleotides in the Dystrophin Gene in Dogs
9:37
Conclusion
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。
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