# VEGF-Mimetic Supramolecular Nanoparticles for Treating Spinocerebellar Ataxia Type 1

> **NIH NIH R61** · NORTHWESTERN UNIVERSITY · 2024 · $401,334

## Abstract

Spinocerebellar ataxia type 1 (SCA1) is a progressive neurodegenerative disease caused by a
polyglutamine expansion in the protein ATXN1. In the course of delineating early mechanisms
underlying neurodegeneration, we made the unexpected discovery that ATXN1 directly regulates the
expression of the angiogenic and neurotrophic cytokine VEGF and that VEGF levels are abnormally
low in the SCA1 mouse brain with pathologic consequences. We have since discovered that delivering
recombinant VEGF is therapeutic in the well-characterized SCA1 knock-in mouse (SCA1154Q/2Q;
Q=glutamine), the most precise existing mouse model of SCA1. However, there are several challenges
with developing recombinant VEGF as a therapy. Here we propose to build upon our recent work
developing a VEGF-mimetic particle that could be readily synthesized with a greater potential to
engage VEGF receptors in both neurons and endothelial cells to maintain neurovascular health in
SCA1. The goal is to engineer a first-in-class nanotherapy for SCA1.

## Key facts

- **NIH application ID:** 10847354
- **Project number:** 5R61NS127141-02
- **Recipient organization:** NORTHWESTERN UNIVERSITY
- **Principal Investigator:** Puneet Opal
- **Activity code:** R61 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $401,334
- **Award type:** 5
- **Project period:** 2023-08-01 → 2026-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10847354

## Citation

> US National Institutes of Health, RePORTER application 10847354, VEGF-Mimetic Supramolecular Nanoparticles for Treating Spinocerebellar Ataxia Type 1 (5R61NS127141-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10847354. Licensed CC0.

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