# Center of Research Translation in Muscular Dystrophy Therapeutic Development

> **NIH NIH P50** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2020 · $1,417,545

## Abstract

Overall CORT
Abstract
The Center for Gene Therapy at The Research Institute of Nationwide Children's Hospital
(RINCH) has a dedicated translational program that targets the muscular dystrophies, with a
particular longstanding interest in developing meaningful therapies for the most common forms,
including Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy
(FSHD). Our Center's goals include unraveling disease pathogenesis and developing new
treatment paradigms that can be translated from the bench to the bedside, and under this
CORT proposal we seek to accelerate this translational process. Project 1 (PI, Paul Martin,
PhD) seeks extend a therapy now entering trials in DMD to other forms of muscular dystrophy,
by applying the overexpression of Galgt2, an enzyme that alters skeletal muscle glycosylation to
boost the expression of proteins that ameliorate disease. Project 2 (PI, Scott Harper, PhD)
explores novel approaches to modulating the expression of the DUX4 gene to treat the relatively
common and debilitating FSHD. Project 3 (PI, Kevin Flanigan, MD) seeks to rapidly translate a
newly discovered mechanism for dystrophin translational control into meaningful therapy for
boys with DMD. All three projects make use of two critical research cores: the Therapeutic Viral
Vector Design and Development Research Core (PI, Louise Rodino-Klapac, PhD) and the
Muscular Dystrophy Cell and Serum Banking Core (PI, Kim McBride, MD). In addition to the
investigators represented here by projects, the proposed CORT represents a larger muscle
disease research base at the Nationwide Children's and the Ohio State University, for which a
well-developed pilot and feasibility program is described. This proposed CORT is consistent
with the mission of NIAMS, which has the goal of finding effective treatments for and to
improving the quality of life of patients with debilitating forms of muscle disease.

## Key facts

- **NIH application ID:** 10016996
- **Project number:** 5P50AR070604-05
- **Recipient organization:** RESEARCH INST NATIONWIDE CHILDREN'S HOSP
- **Principal Investigator:** KEVIN M FLANIGAN
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,417,545
- **Award type:** 5
- **Project period:** 2016-09-14 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10016996

## Citation

> US National Institutes of Health, RePORTER application 10016996, Center of Research Translation in Muscular Dystrophy Therapeutic Development (5P50AR070604-05). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10016996. Licensed CC0.

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