# Clinical and Translational Studies in Muscle Disease

> **NIH NIH K24** · WASHINGTON UNIVERSITY · 2020 · $172,614

## Abstract

The overarching goal of this K24 application is to further my development as a clinical
investigator and support the training of future physician scientists. My patient oriented research
interests relate to understanding the clinical, pathologic, genetic and pathomechanistic
underpinnings of inherited and acquired forms of muscle weakness. With the support of this
grant, I will continue to perform genetic discovery of patients with muscle disease, increase our
biorepository and develop phenotypic assays to understand novel disease pathogenesis. In
addition, I will increase my mentorship responsibilities of graduate students, resident physicians,
fellows and junior faculty within the neuromuscular group and Department of Neurology at
Washington University School of Medicine. Moreover, I will learn valuable skills related to whole
genome sequencing, RNA sequencing and induce pluripotent stem cell differentiation. A K24
grant would protect 50% effort and relieve future clinical and administrative responsibilities. The
two interrelated aims of this proposal are 1) Perform genetic discovery on patients with
unresolved inherited and acquired myopathies using whole genome and RNA sequencing
strategies. 2) Develop and phenotype iPSC derived myoblast models of inherited and acquired
myopathies. These aims will be achieved utilizing our existing biorepository within the
Washington University School of Medicine Neuromuscular Genetics Project and the acquisition
of new patients and patient material. Support through a K24 Midcareer Investigator Award in
Patient-Oriented Research would come at a critical time in my career as I solidify my
independent research program and increase my availability to mentor graduate students, post-
doctoral fellows, residents, neuromuscular fellows and junior faculty in translational myology.
Upon completion of this award, I will have integrated clinical trainees into our existing
translational research infrastructure and created a successful pipeline to generate the next
generation of clinician-scientists focused on muscle diseases.

## Key facts

- **NIH application ID:** 9905490
- **Project number:** 5K24AR073317-03
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** CONRAD C WEIHL
- **Activity code:** K24 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $172,614
- **Award type:** 5
- **Project period:** 2018-07-01 → 2023-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9905490

## Citation

> US National Institutes of Health, RePORTER application 9905490, Clinical and Translational Studies in Muscle Disease (5K24AR073317-03). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9905490. Licensed CC0.

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