# Cell Signaling and Neurodegeneration

> **NIH NIH R01** · UNIVERSITY OF MINNESOTA · 2020 · $393,599

## Abstract

Spinocerebellar ataxia type 1 (SCA1) is one of nine fatal inherited neurodegenerative diseases caused by
expansion of an in-frame CAG trinucleotide repeat. Each repeat tract encodes a stretch of glutamine residues in
the affected protein, in the case of SCA1 the protein is ataxin-1 (ATXN1). Symptoms of SCA1 include loss of
motor coordination and balance, slurred speech, swallowing difficulty, spasticity, and some cognitive
impairment. A characteristic feature of SCA1 pathology is atrophy and eventual loss of Purkinje cells from the
cerebellar cortex. Like many neurodegenerative disorders, SCA1 is typically a late onset disease suggesting
that physiological changes due to aging contribute to the onset of the disease. There is currently no effective
treatment. Thus, identifying signaling pathways and cellular mediators of SCA1 onset and progression remain
a major challenge in the search for therapeutics and is the focus of the research outlined in this application for
continued support. The major aims of this competitive renewal are to further examine the role of ATXN1-S776
phosphorylation by examining the impact of altering ATXN1-S776 phosphorylation on polyQexp ATXN1 toxicity the
brainstem-medulla, and characterize a novel protective pathway activated by a cholecystokinin receptor 1
(Cck1R) agonist in SCA Purkinje cells.

## Key facts

- **NIH application ID:** 9924651
- **Project number:** 5R01NS045667-18
- **Recipient organization:** UNIVERSITY OF MINNESOTA
- **Principal Investigator:** Harry T. Orr
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $393,599
- **Award type:** 5
- **Project period:** 2003-08-15 → 2023-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9924651

## Citation

> US National Institutes of Health, RePORTER application 9924651, Cell Signaling and Neurodegeneration (5R01NS045667-18). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9924651. Licensed CC0.

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