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Embryo genome editing
• Highest efficiency expected in one cell stage zygotes • High efficiency to produce both single mutants (95%)
and double-mutant (70-80%) using direct injection of Cas9 mRNA and sgRNA into zygotes
PCC Genetics - ESHRE BCN 2018
Disease correction. Mouse Model
• Correction of the dystrophin gene (Dmd) mutation in the germline of mdx mice, a model for DMD
• Monitoring of muscle structure and function.
• Genome editing produced genetically mosaic animals containing 2 to 100% correction of the Dmd gene.
The degree of muscle phenotypic rescue in mosaic mice exceeded the efficiency of gene correction, likely reflecting an advantage of the corrected cells and their contribution to regenerating muscle.
Prevention of muscular dystrophy in mice by CRISPR/Cas9–mediated editing of germline DNA . Long,2014
PCC Genetics - ESHRE BCN 2018
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