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Generation of muscular dystrophy model rats with a CRISPR/Cas system.

Authors: Nakamura, K  Fujii, W  Tsuboi, M  Tanihata, J  Teramoto, N  Takeuchi, S  Naito, K  Yamanouchi, K  Nishihara, M 
Citation: Nakamura K, etal., Sci Rep. 2014 Jul 9;4:5635. doi: 10.1038/srep05635.
Pubmed: (View Article at PubMed) PMID:25005781
DOI: Full-text: DOI:10.1038/srep05635

Duchenne muscular dystrophy (DMD) is an X-linked lethal muscle disorder caused by mutations in the Dmd gene encoding Dystrophin. DMD model animals, such as mdx mice and canine X-linked muscular dystrophy dogs, have been widely utilized in the development of a treatment for DMD. Here, we demonstrate the generation of Dmd-mutated rats using a clustered interspaced short palindromic repeats (CRISPR)/Cas system, an RNA-based genome engineering technique that is also adaptive to rats. We simultaneously targeted two exons in the rat Dmd gene, which resulted in the absence of Dystrophin expression in the F0 generation. Dmd-mutated rats exhibited a decline in muscle strength, and the emergence of degenerative/regenerative phenotypes in the skeletal muscle, heart, and diaphragm. These mutations were heritable by the next generation, and F1 male rats exhibited similar phenotypes in their skeletal muscles. These model rats should prove to be useful for developing therapeutic methods to treat DMD.

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CRRD Object Information
CRRD ID: 11040981
Created: 2016-03-18
Species: All species
Last Modified: 2016-03-18
Status: ACTIVE



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RGD is funded by grant HL64541 from the National Heart, Lung, and Blood Institute on behalf of the NIH.