# Myoediting of Duchenne Muscular Dystrophy

> **NIH NIH P50** · UT SOUTHWESTERN MEDICAL CENTER · 2020 · $1,600,891

## Abstract

Project Summary/Abstract for the Overall Component
The techniques of CRISPR/Cas9-mediated genomic editing and the ability to generate induced pluripotent stem
cells (iPSCs) from a sample of a patient’s blood have placed medicine on the brink of a revolution in our ability
to treat, and perhaps even cure, a broad range of genome-based diseases. The overall goal of the UT
Southwestern Wellstone Muscular Dystrophy Specialized Research Center is to improve the treatment provided
to Duchenne muscular dystrophy (DMD) patients by developing a new therapeutic strategy called “myoediting”.
The Center has been built around five integral components. These include: two inter-related research projects
(1) one that will work to optimize the tools for application of CRISPR/Cas9-mediated DMD exon skipping to
permanently restore dystrophin function, and the other (2) that will identify genetic and biomarker associations
with cardiac phenotypes in patients with dystrophinopathies [i.e. DMD and Becker muscular dystrophy (BMD)]
and serve as a primary source for human iPSCs. These projects will be complemented and supported by three
Cores (A) an Administrative Core, that will also direct patient outreach and education, (B) a Myoediting Scientific
Research Resource Core, which will generate DMD/BMD iPSCs from DMD/BMD patients and differentiate them
into iPSC-derived cardiomyocytes as well as house the DMD/BMD Biobank, which will store relevant clinical
data as well as validated guide RNAs for each genetic mutation, and (C) a Training Core, which will enhance the
educational environment in order to recruit, train, and maintain the next generation of transformative investigators
focused on addressing the challenges of muscular dystrophy. We firmly believe myoediting will offer an
innovative therapeutic modality for the treatment of many thousands of DMD patients and offer a long awaited
hope to these patients and their families devastated by DMD.

## Key facts

- **NIH application ID:** 10049124
- **Project number:** 2P50HD087351-06
- **Recipient organization:** UT SOUTHWESTERN MEDICAL CENTER
- **Principal Investigator:** RHONDA BASSEL-DUBY
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,600,891
- **Award type:** 2
- **Project period:** 2015-09-15 → 2025-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10049124

## Citation

> US National Institutes of Health, RePORTER application 10049124, Myoediting of Duchenne Muscular Dystrophy (2P50HD087351-06). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10049124. Licensed CC0.

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