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DOI :
10.3791/57560-v
August 2nd, 2018
Chapters
0:04
Title
1:23
Design and Cloning of gRNAs into the CRISPR Vector
5:51
Intraperitoneal Injection of rAAVrh.74-CRISP into Neonatal mdx Mice
6:38
Analysis of Dystrophin Expression by Immunofluorescence Staining
7:31
Results: rAAV-Mediated Gene Editing Restores Dystrophin Expression in Dystrophic Mice
8:34
Conclusion
在这里, 我们提供了一个详细的协议, 以进行体内心脏基因编辑的小鼠使用重组腺相关病毒 (rAAV) 介导的 CRISPR 交付。本协议为治疗杜氏肌营养不良症营养不良性型心肌病提供了一种有前景的治疗策略, 可用于产后小鼠心脏特异性击倒。
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