Phenotyping an adult zebrafish lamp2 cardiomyopathy model identifies mTOR inhibition as a candidate therapy

Adult zebrafish is an emerging vertebrate model for studying genetic basis of cardiomyopathies; but whether the simple fish heart can model essential features of hypertrophic cardiomyopathy (HCM) remained unknown. Here, we report a comprehensive phenotyping of a lamp2 knockout (KO) mutant. LAMP2 encodes a lysosomal protein and is a causative gene of Danon disease that is characterized by HCM and massive autophagic vacuoles accumulation in the tissues. There is no effective therapy yet to treat this most lethal cardiomyopathy in the young.

Source: Journal of Molecular and Cellular Cardiology - June 18, 2019 Category: Cytology Authors: Alexey V. Dvornikov, Mingmin Wang, Jingchun Yang, Ping Zhu, Tai Le, Xueying Lin, Hung Cao, Xiaolei Xu Source Type: research