# Administrative Core

> **NIH NIH P50** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2020 · $138,352

## Abstract

Administrative Core:
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.
The role of the Administrative Core will be to (1) facilitate scientific integration among these
three projects; (2) oversee the budgetary and reporting aspects of the CORT; (3) develop and
organize the Advisory Board's efforts; and (4) cultivate or identify pilot and feasibility projects for
submission to the NIAMS for funding consideration. On a broader level, the Administrative Core
will be responsible for maximizing the synergy between this CORT and other existing
translational efforts at RINCH, and at the Ohio State University.

## Key facts

- **NIH application ID:** 10017011
- **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:** $138,352
- **Award type:** 5
- **Project period:** 2016-09-14 → 2022-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10017011

## Citation

> US National Institutes of Health, RePORTER application 10017011, Administrative Core (5P50AR070604-05). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10017011. Licensed CC0.

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