# Evaluation of nonviral gene editing systems in the brain assisted by focused ultrasound

> **NIH NIH UH3** · COLUMBIA UNIVERSITY HEALTH SCIENCES · 2022 · $321,741

## Abstract

Supplemental Proposal
Evaluation of nonviral gene editing systems in the brain assisted by focused ultrasound
Abstract
Efficient nonviral gene editing of the brain may help tackle some of the intractable neuronal degenerative
disorders. Compared with viral gene editing, it is more translatable since it should have more manageable
toxicity and immunogenicity. Many gene editing applications may require multiple administrations if there is a
high turnover of the tissue. Viral gene editing systems would be unsuitable because of concerns of
immunogenicity. The major barrier to nonviral gene editing of the brain is the BBB. We previously pioneered
the use of focused ultrasound (FUS) technology to achieve noninvasive, brain-specific delivery of therapeutic
payloads including small molecules, proteins and adeno-associated virus (AAV). This Supplemental proposal
aims to compare the nonviral gene editing systems developed by other SCGE teams. It is expected that these
nonviral gene editing systems have shown promising editing efficiency in tissues other than the brain. The FUS
delivery will be performed at Columbia on Ai9 mice and the brain tissues sent to JAX for independent gene
editing analysis. The final experimental design will be established in consultation with the PIs. This unbiased
comparative study will define the possibilities and limitations of nonviral brain gene editing in the brain assisted
by FUS.

## Key facts

- **NIH application ID:** 10658371
- **Project number:** 3UH3NS115598-04S1
- **Recipient organization:** COLUMBIA UNIVERSITY HEALTH SCIENCES
- **Principal Investigator:** KAM W LEONG
- **Activity code:** UH3 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $321,741
- **Award type:** 3
- **Project period:** 2019-09-01 → 2023-07-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10658371

## Citation

> US National Institutes of Health, RePORTER application 10658371, Evaluation of nonviral gene editing systems in the brain assisted by focused ultrasound (3UH3NS115598-04S1). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10658371. Licensed CC0.

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