Exon skipping
- The use of genetically humanized animal models for personalized medicine approaches
Summary: Personalized medicine approaches benefit from humanized animal models. Here, we outline the usefulness, caveats and considerations for generating and using these models for pre-clinical studies of Duchenne muscular dystrophy.
- CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene
Summary: The authors describe the creation of a mouse strain that reproduces a newly identified deletion mutation in a DMD patient in Russia, and present the characteristics of this new model.