# VCP in myopathy and dementia

> **NIH NIH R01** · WASHINGTON UNIVERSITY · 2022 · $736,659

## Abstract

The overarching goal of R01 renewal is to continue our work on a multisystem degenerative
disorder with muscle weakness and fronto-temporal dementia. VCP pathologies are unified by
ubiquitin and TDP43 inclusions. VCP disease mutations affect multiple cellular process such as
autophagy and endolysosomal trafficking that converge at the lysosome. Recently we have
identified a critical role for VCP in maintaining lysosome integrity that is impaired in the setting of
disease mutatins. We plan to perform the following aims will 1) Evaluate the role of VCP in
lysophagy in neurons and muscle; 2) Evaluate lysosome mediated signaling pathways such as
mTORC1 and TFEB in the brains of mice with VCP disease mutations; 3) Explore the role of
VCP in the endolysosomal escape of proteopathic seeds. Upon completion we will understand
the role of VCP in lysosomal homeostasis and how this is affected by VCP disease mutations in
muscle and neurons.

## Key facts

- **NIH application ID:** 10356903
- **Project number:** 5R01AG031867-14
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** CONRAD C WEIHL
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $736,659
- **Award type:** 5
- **Project period:** 2009-01-01 → 2024-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10356903

## Citation

> US National Institutes of Health, RePORTER application 10356903, VCP in myopathy and dementia (5R01AG031867-14). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10356903. Licensed CC0.

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