Official websites use .gov

A .gov website belongs to an official government organization in the United States.

Secure .gov websites use HTTPS

A lock ( 🔒 ) or https:// means you've safely connected to the .gov website. Share sensitive information only on official, secure websites.

Need Help

Genetic repair of muscular degeneration associated with Duchenne muscular dystrophy

Award Number: R15AR087731
ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 08/01/2026
PERIOD OF PERFORMANCE END DATE: 07/31/2029

Group Awards By:

View Award Description

Genetic repair of muscular degeneration associated with Duchenne muscular dystrophy - Summary Duchenne muscular dystrophy (Dmd) is a severe genetic disorder that causes progressive muscle degeneration, affecting approximately 1 in 5,000 males. It is caused by mutations in the gene encoding dystrophin, a structural protein critical for maintaining muscle fiber integrity. While significant progress has been made with gene therapy and corticosteroids, these treatments focus on skeletal and cardiac muscle while 74% of patients develop life-limiting bladder dysfunction and 68% suffer severe bowel complications. There remains a critical need to understand smooth muscle pathology mechanisms affecting three-quarters of DMD patients. This project investigates smooth muscle dysfunction using C. elegans, the first platform enabling systematic cross-tissue comparison within individual animals. Building on discoveries from our prior NIH-funded work, we focus on smooth muscle systems controlling gastrointestinal function. Our preliminary data show that corticosteroids rescue striated but not smooth muscle dysfunction, directly mirroring clinical observations where patients show improved locomotion but persistent gastrointestinal problems. We use C. elegans alongside human smooth muscle cell validation systems. In Aim 1, we will characterize smooth muscle dysfunction using behavioral analysis, multi-compartment calcium imaging, and transcriptomic profiling. In Aim 2, we will evaluate therapeutic efficacy across muscle types, testing microdystrophin constructs and corticosteroids for differential rescue patterns. This work seeks to define smooth muscle pathology mechanisms that remain therapeutically unaddressed. By establishing the first cross-tissue platform for smooth muscle DMD research, it will identify tissue-specific therapeutic requirements for comprehensive treatments. Additionally, it will provide research training for 70+ undergraduate students through direct hands-on involvement in all research phases, preparing the next generation of scientists with cutting-edge microscopy, behavioral, and translational research techniques while fulfilling R15 AREA objectives of strengthening the research environment at undergraduate-focused institutions.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $544,734 )
2026	2026	BOARD OF TRUSTEES OF ILLINOIS STATE UNIVERSITY	CAMPUS BOX 1100	NORMAL	IL	61790	MCLEAN	USA	Arthritis, Musculoskeletal and Skin Diseases Research	000	1	7/15/2026	NEW	$544,734
														Subtotal = $544,734

Grand Total All Awards = $544,734

Top

All Categories

About

Search

Reports

Data Submission

Award Information

Genetic repair of muscular degeneration associated with Duchenne muscular dystrophy

Award Number: R15AR087731

ORGANIZATION: NATIONAL INSTITUTE OF ARTHRITIS & MUSCULOSKELETAL & SKIN DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 08/01/2026

PERIOD OF PERFORMANCE END DATE: 07/31/2029

Federal Websites

Department of Health & Human Services

HHS Operating Divisions

HHS Staff Divisions

Download A Document Viewer