# Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center - Seattle

> **NIH NIH P50** · UNIVERSITY OF WASHINGTON · 2024 · $1,674,346

## 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 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) identify
optimized AAV variants for skeletal and cardiac muscle, modify micro-dystrophin sequences to diminish
immunogenicity and increase function, and test split intein vector strategies for delivering more potent
dystrophin constructs to skeletal and cardiac muscle; (b) will apply parallel AAV-mediated methods to achieve
the suppression of human DUX4 in a mouse model of FSHD and in a large animal porcine model of FSHD,
extending the use of similar vector technologies as a general delivery platform for dominant muscular
dystrophies and opening new therapeutic opportunities for FSHD. Aim 2 (Project 2) will establish
facioscapulohumeral dystrophy clinical trial foundations. Studies in this aim will (a) perform clinical and MRI
assessment in a long-term extension of the prior Seattle Wellstone FSHD cohorts and apply newer methods
of MRI data analysis to generate a multi-year composite dataset of FSHD disease progression for correlations
with disease progression; (b) perform a dose-escalation safety and tolerability study of a therapeutic candidate
that will incorporate the most current MRI and molecular characteristics to assess their performance for the
design of future clinical trials; and (c) perform functional, MRI, and molecular characterization of a new porcine
model of FSHD to determine its utility as a preclinical model for human studies. 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, and Training) 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, provide training of the next generation of scientific and clinical researchers in muscular
dystrophy and conduct outreach and educational activities. Together, these aims will achieve the overall goal
to develop the reagents, measurements, clinical trials methods, and clinical trials infrastructures to speed the
development of effective therapies for DMD and FSHD, and to bring muscular dystrophy clinical trials and
therapies to the families of the Northwest and beyond.

## Key facts

- **NIH application ID:** 10922746
- **Project number:** 5P50AR065139-11
- **Recipient organization:** UNIVERSITY OF WASHINGTON
- **Principal Investigator:** JEFFREY S CHAMBERLAIN
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $1,674,346
- **Award type:** 5
- **Project period:** 2014-05-07 → 2028-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10922746

## Citation

> US National Institutes of Health, RePORTER application 10922746, Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center - Seattle (5P50AR065139-11). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10922746. Licensed CC0.

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