期刊
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
卷 23, 期 5, 页码 -出版社
MDPI
DOI: 10.3390/ijms23052898
关键词
dystrophin; mutant; CRISPR; Cas9; dog; somatic cell nuclear transfer
资金
- Institute for Basic Science [IBS-R021-D1, 550-20200076]
- Research Institute for Veterinary Science
- BK21 plus program
A dystrophin mutant dog was successfully generated using the CRISPR/Cas9 system and somatic cell nuclear transfer in this study. This model can be used to study human dystrophinopathy and aid in the development of gene therapy drugs for dogs and humans.
Dystrophinopathy is caused by mutations in the dystrophin gene, which lead to progressive muscle degeneration, necrosis, and finally, death. Recently, golden retrievers have been suggested as a useful animal model for studying human dystrophinopathy, but the model has limitations due to difficulty in maintaining the genetic background using conventional breeding. In this study, we successfully generated a dystrophin mutant dog using the CRISPR/Cas9 system and somatic cell nuclear transfer. The dystrophin mutant dog displayed phenotypes such as elevated serum creatine kinase, dystrophin deficiency, skeletal muscle defects, an abnormal electrocardiogram, and avoidance of ambulation. These results indicate that donor cells with CRISPR/Cas9 for a specific gene combined with the somatic cell nuclear transfer technique can efficiently produce a dystrophin mutant dog, which will help in the successful development of gene therapy drugs for dogs and humans.
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