# Muscular Dystrophy Cell Line and Serum Banking Core

> **NIH NIH P50** · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · 2024 · $294,735

## Abstract

Muscular Dystrophy Cell Line and Serum Banking Core
ABSTRACT
The over-arching theme for this MDSRC grant application is to accelerate the translation of novel genetic
therapies from the bench into the clinic for individuals with muscular dystrophy. A significant bottleneck in
studying pathogenic variants or their correction is the limited proliferative capacity of muscle cells from
muscular dystrophy patients, and the process of obtaining cells (muscle biopsy) is invasive. A technique using
lentiviral vectors for both hTERT and MyoD delivery to dermal fibroblasts derived from skin biopsy to create
myogenic fibroblasts (hereafter called FibroMyoD) will overcome this bottleneck. Through a previous P50
grant we have been very successful creating and banking several hundred fibroblast and FibroMyoD cell
lines. The overall objective of the Muscular Dystrophy Cell Line and Serum Banking Core (MD-CLSB Core)
will be to continue banking human primary and immortalized cell lines and improve on the method to create
myoblasts that will support each of the MDSRC Projects, as well as for serum and plasma banking for
exploratory and collaborative projects. The overall objective will be addressed in two specific aims. Aim 1 will
expand our unique cell line resource from muscular dystrophy subjects by banking dermal fibroblasts and
creating FibroMyoD cell lines and continue banking of serum samples from muscular dystrophy subjects. In Aim
2 we will develop an improved transdifferentiation protocol to generate mature myofibers from the FibroMyoD
lines that more closely mimic mature myofibers. We will use novel 3D microenvironments in combination with
new transgene delivery to convert dermal fibroblasts to FibroMyoD, different media formulations and extracellular
matrix composition to create a more mature myoblast. This MD-CLSB Core will leverage the existing expertise
of the Nationwide Children’s Hospital (NCH) Research Institute Cell Line Core, codirected by Drs. Flanigan, MD
and Wein, Ph.D. This institutionally supported shared resource has been creating and banking cell lines for over
15 years. The current proposal involves a greatly increased demand on the existing NCH Cell Line Core, and
establishment of the MD-CLSB Core will provide the additional resources needed to serve as a resource to both
the NCH/OSU and the broader muscle research community. The cell lines generated, and serum samples
stored is and will continue to be a valuable resource for the projects of the proposed MDSRC, and the broader
muscular dystrophy research community, in direct alignment with the MDSRC mission.

## Key facts

- **NIH application ID:** 10992525
- **Project number:** 1P50HD117373-01A1
- **Recipient organization:** RESEARCH INST NATIONWIDE CHILDREN'S HOSP
- **Principal Investigator:** Nicolas Wein
- **Activity code:** P50 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $294,735
- **Award type:** 1
- **Project period:** 2024-08-15 → 2028-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10992525

## Citation

> US National Institutes of Health, RePORTER application 10992525, Muscular Dystrophy Cell Line and Serum Banking Core (1P50HD117373-01A1). Retrieved via AI Analytics 2026-06-12 from https://api.ai-analytics.org/grant/nih/10992525. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*
