# Advancing RNA Therapeutics for Huntington’s Disease

> **NIH NIH U01** · UNIV OF MASSACHUSETTS MED SCH WORCESTER · 2020 · $1,436,316

## Abstract

ABSTRACT
The cause of Huntington’s disease is an increase in the trinucleotide CAG repeat from under
36 repeats to 36 or greater repeats. The mode for the number of repeats is 42, and most
patients have between 40 and 45. The disease generally starts between ages 30 and 40,
with onset and progression of impaired cognition, depression, and aberrant movement. The
genetics is autosomal dominant. Lowering mutant huntingtin in HD animal models delays
onset of disease or mitigates the severity of disease. We use advanced, modified, di-
branched siRNA to lower mutant huntingtin, by invoking RNA interference in brain. Our
advanced siRNA achieves therapeutic advantages: spread throughout the brain in non-
human primate and long-term huntingtin lowering after a single administration into the
cerebrospinal fluid. The siRNA will be optimized in structure for safety and preliminary
results will be secured. The goal of this CREATE proposal is to set the stage for promising
therapeutics for treatment of Huntington’s disease. Similar treatments could be applicable to
other autosomal dominant neurological disorders.

## Key facts

- **NIH application ID:** 9872591
- **Project number:** 1U01NS114098-01
- **Recipient organization:** UNIV OF MASSACHUSETTS MED SCH WORCESTER
- **Principal Investigator:** NEIL ARONIN
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $1,436,316
- **Award type:** 1
- **Project period:** 2020-02-01 → 2025-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9872591

## Citation

> US National Institutes of Health, RePORTER application 9872591, Advancing RNA Therapeutics for Huntington’s Disease (1U01NS114098-01). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9872591. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*