Drug intelligence / Profile preview

calsarcin-1

Development stage
Preclinical
Lead developer
Yale University
Modality
Gene Therapies
Administration
Intravenous, Intramuscular
01

Overview

Calsarcin-1, also known as myozenin-2, is a Z-disc-associated protein that plays a critical role in maintaining the structural integrity of the cardiac sarcomere and modulating calcineurin signaling. In familial hypertrophic cardiomyopathy (HCM), calsarcin-1 levels are often depleted, leading to the destabilization of the muscle LIM protein (MLP)/Z-disc complex and subsequent cardiomyocyte hypertrophy. Therapeutic approaches under investigation involve the use of viral vectors to achieve ectopic expression of calsarcin-1, which has been shown to rescue the hypertrophic phenotype, reduce cell size, and normalize levels of atrial natriuretic factor (ANF) and brain natriuretic peptide (BNP) in patient-specific induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs).

Other names
myozenin-2myozenin2myozenin 2MYOZ2MYOZ-2MYOZ 2
02

Targets

CSRP3 (Cysteine and glycine-rich protein 3 (cardiac LIM protein))PPP3CA (Protein phosphatase 3 catalytic subunit alpha)ACTN2 (Alpha-actinin-2)

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