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Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact.
Muscle tissue specification, development and function in zebrafish are highly conserved with other vertebrates, including humans. In addition, the impact of disease and therapeutics can be studied much earlier due to its fast development. Transparent and accessible embryos allow for non-invasive tissue analysis, immunohistochemistry and automated locomotion monitoring.
DMD model is designed for fast and high-throughput efficacy evaluation of candidate therapeutic compounds. This model allows identifying compounds that prevent muscle degeneration and the subsequent locomotor alterations.
High conservation of zebrafish muscles with vertebrates and humans.DMD muscle damage is already evident in embryos, which allows early studies of disease impact, and therefore, earlier drug efficacy testing.Transparent embryos allow the optical birefringence-based evaluation of muscular integrity.Automated locomotion monitoring highlights muscle functionality impairment rapidly.
High conservation of zebrafish muscles with vertebrates and humans.
DMD muscle damage is already evident in embryos, which allows early studies of disease impact, and therefore, earlier drug efficacy testing.
Transparent embryos allow the optical birefringence-based evaluation of muscular integrity.
Automated locomotion monitoring highlights muscle functionality impairment rapidly.
Early-life Sapje dystrophin-null mutant embryos are exposed to the molecule of interest in order to determine mortality/teratogenicity and muscle integrity dose-effect curves. Muscle integrity is evaluated through optical birefringence, an optic phenomenon resulting from the diffraction of polarized light through the pseudo-crystalline array of the muscle sarcomeres. Zebrafish healthy muscles can be detected as a bright area in an otherwise dark background. Muscle damage, as seen in Sapje mutant, can be detected by a reduction in the birefringence as shown in Figure 1. Phenotypic analysis must be coupled with genotypic homozygous confirmation to determine the percentage of homozygotes that recover the dystrophic phenotype after treatment.