# Wellstone Muscular Dystrophy Specialized Research Center (Seattle)

> **NIH NIH P50** · UNIVERSITY OF WASHINGTON · 2021 · $1,518,858

## Abstract

Overall Summary/Abstract: The major theme of the Seattle Wellstone center is to improve therapeutic
approaches to muscular dystrophies by identifying and overcoming the emerging new barriers to successful
clinical trials in muscular dystrophies. The overall goal is to develop the reagents, measurements, clinical
trials methods, and clinical trials infrastructures to speed the development of effective therapies for Duchene
muscular dystrophy (DMD) and FSHD, and to bring muscular dystrophy clinical trials and therapies to the
families of the Northwest. The specific aims and objectives are to breach the major barriers to successful
therapeutic clinical trials in muscular dystrophies in the Northwest and nationwide. Aim 1 (Project 1) will
conduct translational and pre-clinical studies of muscular dystrophy gene therapy. Studies in this aim will (a)
improve the function of the micro-dystrophin gene, with a focus on cardiac muscle, and identify therapies
that act to improve the functional benefit conferred by micro-dystrophin; (b) adapt these AAV-mediated
delivery methods to achieve the suppression of human DUX4 in a preclinical mouse model of FSHD; and
(c) establish a clinical trials readiness in Seattle for participation in AAV-mediated therapeutic trials in DMD.
Aim 2 (Project 2) will enhance facioscapulohumeral dystrophy (FSHD) clinical trial foundations. The prior
funding period established correlations between functional assessments, MRI characteristics, and molecular
markers in FSHD. Studies in this aim will: (a) determine whether the correlation between candidate
molecular biomarkers and MRI characteristics identified in the prior funding period predict functional
progression; (b) determine whether the correlation and predictive value of the candidate molecular
biomarkers and MRI characteristics identified in the first cohort can be validated in an independent cohort;
and (c) determine the molecular phenotype of the infiltrating mononuclear cells in areas of inflammation and
whether this represents an oligoclonal T-cell response. Aim 3 (Cores A, B, C) will administer, provide
resources for scientific research, and train future muscular dystrophy scientific and clinical researchers. The
Center cores (Administrative, Scientific Research Resource, and Training Cores) will provide support and
oversight of all activities, provide necessary biological resources to achieve the goals of the Center and
serve as a national resource, and provide training of the next generation of scientific and clinical
researchers in muscular dystrophy. Together, these aims will achieve the overall goal to bring muscular
dystrophy clinical trials and therapies to the families of the Northwest and the nation.

## Key facts

- **NIH application ID:** 10248342
- **Project number:** 5P50AR065139-08
- **Recipient organization:** UNIVERSITY OF WASHINGTON
- **Principal Investigator:** JEFFREY S CHAMBERLAIN
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $1,518,858
- **Award type:** 5
- **Project period:** 2014-05-07 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10248342

## Citation

> US National Institutes of Health, RePORTER application 10248342, Wellstone Muscular Dystrophy Specialized Research Center (Seattle) (5P50AR065139-08). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10248342. Licensed CC0.

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