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Genetic therapy for mice with Duchenne muscular dystrophy

Description
Adenovirus carrying Ca59 and gRNA sequences gRNA target sites Permanent Ca59 gRNAs exon skipping Gene lacking exon 23 / \/ \/ l Transcription mRNA lackin 3 exon 2% _ Inject intramuscularly _ into dmd mutant mouse 1 Translation Reading frame restored in protein lacking exon 23
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