This study applies targeted Cas9-based gene insertion strategies for the correction of full-length dystrophin in a pre-clinical humanized mouse model of Duchenne muscular dystrophy. Following intramuscular or intravenous delivery, full-length dystrophin is restored in skeletal and cardiac muscle.
Overall design
High-throughput unbiased sequencing to characterize and quantify gene-editing outcomes in genomic DNA and cDNA transcripts of treated mice.
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