Key result
Introduction of antisense morpholino oligonucleotides restored normal splicing of ryr1b and recovered swimming in zebrafish relatively relaxed mutants.
Population
Zebrafish embryos (wild-type and relatively relaxed mutants)
Comparison
Antisense morpholino oligonucleotides targeting… vs Untreated relatively relaxed mutants and…
Design
Preclinical
Authors
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May guide preclinical antisense therapy development for RYR1 myopathies; leaves open human translation.
Zebrafish relatively relaxed mutants provide a useful preclinical model for understanding the development and physiology of multi-minicore disease caused by RYR1 mutations.
Hirata et al. (2007) studied Multi-minicore disease (MmD). Antisense morpholino oligonucleotides vs. Untreated mutants was evaluated on Swimming recovery. Introduction of antisense morpholino oligonucleotides restored normal splicing of ryr1b and recovered swimming in zebrafish relatively relaxed mutants.
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