Systematic in vivo candidate evaluation uncovers therapeutic targets for LMNA dilated cardiomyopathy and risk of Lamin A toxicity.

ABSTRACT:

Background

Dilated cardiomyopathy (DCM) is a severe, non-ischemic heart disease which ultimately results in heart failure (HF). Decades of research on DCM have revealed diverse aetiologies. Among them, familial DCM is the major form of DCM, with pathogenic variants in LMNA being the second most common form of autosomal dominant DCM. LMNA DCM is a multifactorial and complex disease with no specific treatment thus far. Many studies have demonstrated that perturbing candidates related to various dysregulated pathways ameliorate LMNA DCM. However, it is unknown whether these candidates could serve as potential therapeutic targets especially in long term efficacy.

Methods

We evaluated 14 potential candidates including Lmna gene products (Lamin A and Lamin C), key signaling pathwa

SUBMITTER: Tan CY 

PROVIDER: S-EPMC10577912 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature