Key result
Zebrafish models with filamin C mutations or knockdowns exhibited fiber dissolution and protein aggregates similar to human filamin-related myofibrillar myopathies.
Population
Zebrafish models (stretched out, a zebrafish filamin Cb mutant, and targeted knockdown of zebrafish filamin…
Design
Preclinical
Authors
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Establishes zebrafish filamin C models for myofibrillar myopathy; extends mechanistic research but leaves clinical translation open pending validation.
Zebrafish models with filamin C knockdown successfully replicate the myopathology of filamin-related myofibrillar myopathy, providing a new in vivo model for studying the disease mechanism and potential therapies.
Ruparelia et al. (2012) studied filamin-related myofibrillar myopathy. Zebrafish filamin Cb mutation (sot) and filamin Ca knockdown was evaluated on Fiber dissolution and formation of protein aggregates. Zebrafish models with filamin C mutations or knockdowns exhibited fiber dissolution and protein aggregates similar to human filamin-related myofibrillar myopathies.
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