# Evaluating and Targeting RNA granules in neurodegenerative diseases

> **NIH NIH R56** · UNIVERSITY OF CALIFORNIA, SAN DIEGO · 2020 · $661,492

## Abstract

PROJECT SUMMARY
A hallmark of many neurodegenerative diseases is the presence of abnormal protein aggregates in neurons of
the brain and spinal cord. Two related diseases in which there has been rapid progress in understanding the
origins and consequences of such protein inclusions are Frontotemporal Dementia (FTD) and Amyotrophic
Lateral Sclerosis (ALS). FTD is characterized by progressive behavioral, cognitive, language and motor
symptoms and an average life expectancy of 7-13 years from diagnosis. ALS first manifests as peripheral
muscle weakness but progresses rapidly and is usual fatal due to respiratory failure within 2-5 years from
diagnosis. It has become clear that FTD and ALS are closely related diseases with an overlapping spectrum of
symptoms. In recent years, through work by us and others, dynamic ribonucleoprotein (RNP) assemblies
known as stress granules (SGs) have been implicated in the formation of TDP-43 inclusions, and many
ALS/FTD-causing mutations have been shown to affect SG behavior. Compelling recent studies have shown
that aggregation of proteins into SGs may also be central to the etiology of tauopathies, including Alzheimer’s
disease. Using models of neurodegeneration in stem-cell derived cortical neurons and in flies, the research
proposed in this application is designed to fundamentally enhance our understanding of how SGs are related
to mutant-dependent, and to develop and evaluate novel strategies for therapeutically targeting abnormal
granules. Our ambitious study will lay the foundation for a novel paradigm to study and to potentially treat
dementia and related neurodegenerative diseases.

## Key facts

- **NIH application ID:** 10239754
- **Project number:** 1R56AG069098-01
- **Recipient organization:** UNIVERSITY OF CALIFORNIA, SAN DIEGO
- **Principal Investigator:** Eugene Wei-Ming Yeo
- **Activity code:** R56 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $661,492
- **Award type:** 1
- **Project period:** 2020-09-30 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10239754

## Citation

> US National Institutes of Health, RePORTER application 10239754, Evaluating and Targeting RNA granules in neurodegenerative diseases (1R56AG069098-01). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10239754. Licensed CC0.

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