Why the study?
Little is known about the biological function, causal relationship, and mechanisms underlying MYOM1-related myopathies in the heart, and no MYOM1 knockout model previously existed.
Population
Human embryonic stem cell-derived cardiomyocytes
Comparison
MYOM1 knockout vs control
Design
In vitro gene-editing preclinical study
Authors
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Provides human cardiomyocyte model of MYOM1 deficiency; leaves open clinical relevance and therapeutic targeting of myomesin-1.
Establishment of a MYOM1 knockout human cardiomyocyte model reveals that myomesin-1 deficiency leads to myocardial atrophy and impairs sarcomere assembly.
Hang et al. (2021) studied this question.
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