# Muscular Dystrophy Specialized Research Center

> **NIH NIH P50** · UNIVERSITY OF IOWA · 2021 · $1,508,480

## Abstract

OVERALL SUMMARY
The overall goal of the University of Iowa Wellstone Muscular Dystrophy Specialized Research Center
(MDSRC) is to perform research on the various muscular dystrophies that arise from abnormal processing of
the dystroglycan protein (dystroglycanopathies). The Center will achieve this overall goal by conducting basic
and translational research in dystroglycanopathy patients, patient-derived biosamples, and mouse models. Our
MDSRC application is composed of two projects and three cores, all of which are directed by investigators with
a proven track record of excellence and collaboration in basic, translational and clinical research. Project 1
(Campbell) will investigate the cellular and molecular mechanisms that underly dystroglycanopathies by
evaluating the relationship between α-dystroglycan (α-DG) matriglycan length and its laminin-binding
properties in control and dystroglycanopathy patient fibroblasts and muscle biopsies. Additional studies will
define the requirements for matriglycan synthesis as regulated by protein-protein and protein-sugar
interactions. A dystroglycanopathy mouse model will be utilized to identify novel pathophysiologic mechanisms
and determine the structure and laminin binding properties required to improve muscle function. Project 2
(Mathews) will refine the natural history of FKRP-related dystroglycanopathy derived from an established,
unique cohort of patients by expanding the number of clinical sites evaluating patients and by extending the
natural history study to patients with more advanced clinical severity. Extended follow up of non-FKRP
genotypes will identify cohorts that share similar rates of motor progression who might be studied together in
gene non-specific clinical trials. Candidate proteomic biomarkers in blood or urine for the full spectrum of
dystroglycanopathy genotypes will be validated and related to disease status. Core A (Campbell and Moore) is
an administrative core that will coordinate the activities within and outside the Center, as a means to promote
an interactive and collaborative research environment, and to engage patients in muscular dystrophy research.
Core B (Moore), a Muscle-Tissue/Cell-Culture/Diagnostics Core, will support Projects 1 and 2, serve as a
national tissue and cell-culture resource for research, provide specialized diagnostic testing for a wide range of
muscular dystrophies, and maintain the infrastructure needed to evaluate muscle biopsies in support of clinical
trials. Finally, Core C (Mathews and Campbell) will coordinate our Training initiatives. Among the support this
Core provides will be fellowships enabling two medical students per year to perform research in the Center and
to participate in the care of patients alongside Dr. Mathews, with basic science training opportunities also
provided to a postdoctoral fellow and an undergraduate research fellow. The highly integrated cores and
projects of this Center will accelerate the tempo of discovery in pre...

## Key facts

- **NIH application ID:** 10238928
- **Project number:** 5P50NS053672-17
- **Recipient organization:** UNIVERSITY OF IOWA
- **Principal Investigator:** KEVIN P. CAMPBELL
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,508,480
- **Award type:** 5
- **Project period:** 2005-06-08 → 2025-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10238928

## Citation

> US National Institutes of Health, RePORTER application 10238928, Muscular Dystrophy Specialized Research Center (5P50NS053672-17). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10238928. Licensed CC0.

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