# Astrocyte dysfunction in Spinocerebellar ataxia type 1 (SCA1)

> **NIH NIH R01** · UNIVERSITY OF MINNESOTA · 2024 · $454,999

## Abstract

ABSTRACT
 The goal to effectively treat neurodegenerative disorders requires an understanding not only of neuronal
dysfunction, but also of dysfunction of non-neuronal cells that can initiate and contribute to neuronal pathology.
Spinocerebellar ataxia type 1 (SCA1) is a fatal, dominantly inherited neurodegenerative disease caused by the
abnormal expansion of CAG repeats in the Ataxin 1 (ATXN1) gene. SCA1 patients suffer from progressive
neuronal degeneration and reactive astrogliosis, especially in the cerebellum, leading to motor deficits. Despite
intense research focus on the disease mechanisms in cerebellar neurons, there are no effective therapies
available to cure, delay or ameliorate SCA1.
 Astrocytes are brain cells that play fundamental roles in nearly all aspects of neuronal and brain
function. Recently, using single-nuclei RNA sequencing we demonstrated that cerebellar astrocytes express
Atxn1 at a level comparable to neurons (Borgenheimer et al., 2022). This raises a question of how mutant
ATXN1 in astrocytes impacts their function, contributes to neuronal pathology, and leads to SCA1-like disease
outcomes. To fill this knowledge gap, we aim to determine the molecular and cellular mechanisms though
which mATXN1 affects cerebellar astrocytes, and how astrocyte dysfunctions contribute to the behavioral and
pathological changes that are observed in SCA1 disease onset and progression.

## Key facts

- **NIH application ID:** 10984567
- **Project number:** 1R01NS134916-01A1
- **Recipient organization:** UNIVERSITY OF MINNESOTA
- **Principal Investigator:** Marija Cvetanovic
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $454,999
- **Award type:** 1
- **Project period:** 2024-07-01 → 2029-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10984567

## Citation

> US National Institutes of Health, RePORTER application 10984567, Astrocyte dysfunction in Spinocerebellar ataxia type 1 (SCA1) (1R01NS134916-01A1). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10984567. Licensed CC0.

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