# Improving Focused Ultrasound Mediated Viral Gene Therapy Delivery

> **NIH NIH R01** · UT SOUTHWESTERN MEDICAL CENTER · 2024 · $819,998

## Abstract

Project Summary: The inability to distribute gene products broadly throughout the CNS is a major limitation of current
gene delivery methods for brain disease. Employing white matter connectivity to map brain networks is a powerful, well-
established method in laboratory studies, but has not been exploited clinically for therapeutic delivery. The central goal
of this proposal is to advance a focused ultrasound (FUS)-based gene therapy delivery strategy – “circuit focused
ultrasound” (CIFUS) – that utilizes brain connectivity to widely target interconnected brain regions. Our approach
overcomes several longstanding obstacles that have thwarted the tremendous promise of gene therapy for central
nervous system (CNS) diseases. This proposal will advance the development of CIFUS and test the hypothesis that this
approach can functionally rescue key phenotypes in models of a degenerative and a circuit-based brain disease. We will
use a mouse model of Alzheimer disease with tau pathology that spreads across multiple interconnected brain regions
during maturation and aging.6,7 We will use mouse models of dystonia that recapitulates motor circuit dysfunction8,9 and
neurodevelopmental neurodegeneration8,10. Successful completion of this proposal will advance development of a novel
method of widespread, targeted gene delivery that will empower basic neuroscience research and lay the groundwork
for human trials.

## Key facts

- **NIH application ID:** 10881223
- **Project number:** 1R01NS127900-01A1
- **Recipient organization:** UT SOUTHWESTERN MEDICAL CENTER
- **Principal Investigator:** Bhavya Ramesh Shah
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $819,998
- **Award type:** 1
- **Project period:** 2024-06-01 → 2029-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10881223

## Citation

> US National Institutes of Health, RePORTER application 10881223, Improving Focused Ultrasound Mediated Viral Gene Therapy Delivery (1R01NS127900-01A1). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10881223. Licensed CC0.

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