Inhibition of myostatin improves muscle atrophy in oculopharyngeal muscular dystrophy (OPMD).

ABSTRACT:

Background

Oculopharyngeal muscular dystrophy (OPMD) is a late-onset muscle disease affecting one per 80 000 of the general population characterized by profound dysphagia and ptosis, and limb weakness at later stages. Affected muscles are characterized by increased fibrosis and atrophy. Myostatin is a negative regulator of muscle mass, and inhibition of myostatin has been demonstrated to ameliorate symptoms in dystrophic muscles.

Methods

In this study, we performed a systemic delivery of a monoclonal antibody to immunologically block myostatin in the A17 mouse model of OPMD. The mice were administered a weekly dose of 10 mg/kg RK35 intraperitonially for 10 weeks, following which histological analyses were performed on the samples.

Results

This treatment significantly

SUBMITTER: Harish P 

PROVIDER: S-EPMC6818462 | biostudies-literature | 2019 Oct

REPOSITORIES: biostudies-literature