# Development of a nanoparticle-based gene editing technology for neurological applications

> **NIH NIH UH3** · OHIO STATE UNIVERSITY · 2022 · $499,996

## Abstract

Abstract
This supplement proposal focuses on investigating if CRISPR Cas9 ribonucleoproteins (RNPs) complexed to
cell-penetrating peptides (CPPs) can rescue mice from Huntington’s disease, using the R6/2 mouse model.
These experiments are based upon our recent studies demonstrating that CPPs can efficiently transfect CRISPR
Cas9-RNPs neurons in the striatum after an intracranial injection using convection enhanced delivery (CED).
We have selected the 2/6 mouse model for these studies because this model has recently been rescued with
Cas9 based genome editing, using AAV delivered SaCas9. In this proposal we will build on our Preliminary
Studies and will inject Cas9-RNP complexed to CPPs into the striatum and thalamus, via CED, and will
investigate if we can edit the huntingtin (HTT) gene to rescue mice from Huntington’s disease. If successful,
these experiments will identify the levels of genome editing needed in the brain to generate therapeutic outcomes
in the Huntington’s animal model.

## Key facts

- **NIH application ID:** 10619048
- **Project number:** 3UH3NS115599-04S1
- **Recipient organization:** OHIO STATE UNIVERSITY
- **Principal Investigator:** Krystof S Bankiewicz
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $499,996
- **Award type:** 3
- **Project period:** 2019-09-15 → 2024-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10619048

## Citation

> US National Institutes of Health, RePORTER application 10619048, Development of a nanoparticle-based gene editing technology for neurological applications (3UH3NS115599-04S1). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/10619048. Licensed CC0.

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