# MOLECULAR MECHANISMS OF DYSTROPHIC CARDIOMYOPATHY

> **NIH NIH R01** · BOSTON CHILDREN'S HOSPITAL · 2020 · $595,711

## Abstract

MOLECULAR MECHANISMS OF DYSTROPHIC CARDIOMYOPATHY
Abstract
Cardiovascular diseases continue to be a leading cause of death and disability in USA. Duchenne Muscular
Dystrophy (DMD) is a genetic disorder caused by mutations in the dystrophin gene that affects the structure and
function of both cardiac and skeletal muscles. Heart failure has become a leading cause of fatalities in DMD
patients. Our goal is to understand the molecular mechanisms underlying dystrophic cardiomyopathy, and to
develop novel therapeutic strategies to treat this disease.
 We have first discovered CIP as a novel cardiomyocyte-enriched protein and we showed CIP is dynamically
regulated in hypertrophic and dilated hearts. CIP physically interacts with dystrophin, mutation of which causes
DMD. Importantly, we observed that CIP overexpression protects DMD mice (Mdx) from cardiomyopathy. These
exciting findings have identified CIP as a novel component of the dystrophic cardiomyopathy pathway. In this
study, we will define the molecular nature of CIP action and test the therapeutic potential of this protein in
protecting the heart from developing dystrophic cardiomyopathy. More specifically, we will:
1) define the functional mechanism of CIP in dystrophic cardiomyopathy and heart failure;
2) test the therapeutic potential of CIP in dystrophic cardiomyopathy;
3) test the hypothesis that CIP mediates the oxidative stress signaling pathway in dystrophic cardiomyopathy.
 Studies proposed in this application will yield new insights into the pathogenesis of cardiomyopathy in DMD.
Furthermore, we will demonstrate that therapy is a viable strategy to treat dystrophic cardiomyopathy in both
murine and human disease models.

## Key facts

- **NIH application ID:** 9983786
- **Project number:** 5R01HL149401-02
- **Recipient organization:** BOSTON CHILDREN'S HOSPITAL
- **Principal Investigator:** Da-Zhi Wang
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $595,711
- **Award type:** 5
- **Project period:** 2019-08-01 → 2023-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9983786

## Citation

> US National Institutes of Health, RePORTER application 9983786, MOLECULAR MECHANISMS OF DYSTROPHIC CARDIOMYOPATHY (5R01HL149401-02). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/9983786. Licensed CC0.

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