# Nanoscience-Inspired Acoustofluidic Assembly Lines for Gene and Cellular Therapies

> **NIH NIH DP5** · UNIVERSITY OF CALIFORNIA LOS ANGELES · 2023 · $390,000

## Abstract

Project Summary
Stem cell-based gene therapies that leverage gene-editing approaches to address disease-causing mutations
are emerging as viable medical interventions across a variety of pathologies. Current viral-vector-based and
non-viral gene-transfer methods of delivering gene editing machinery, which involve either chemical or energetic
disruption of cell membranes, are used routinely in laboratory settings, but fall short when scaled up for clinically
relevant applications targetting the manufacture of therapeutic cell products. New methods that enable efficient,
rapid, safe, and economical delivery of gene editing packages are needed to support the infrastructures that will
be required to translate these gene therapies broadly for application in patient care. Our solution to this critical
unmet need leverages innovations in gene editing and nanotechnology to render cell membranes transiently
porous, enabling intracellular delivery of biomolecular cargoes. We will design and apply new methods that use
acoustic waves generated within microfluidic systems (i.e., acoustofluidics) to mechanically disrupt cell
membranes, facilitating the rapid and efficient delivery of CRISPR/Cas9 gene-editing components that are
packaged into supramolecular nanocarriers. We use sickle cell disease, one of the most common
hemoglobinopathies worldwide, as an initial clinical target for evaluating the proposed platform as it arises from
a well-defined genetic mutation that can be targetted for site-specific correction in hematopoietic stem cells with
gene editing systems such as CRISPR/Cas9. Successful execution of this research will pave the way for
technologies that enable rapid and sustainable processing of stem cell-based gene therapies at clinically-
applicable doses – effectively establishing scalable, good manufacturing practice-compatible assembly lines for
manufacturing gene modified therapeutic cell products to treat a wide variety of disesases and will streamline
the clinical deployment of future cellular therapies.

## Key facts

- **NIH application ID:** 10693313
- **Project number:** 5DP5OD028181-05
- **Recipient organization:** UNIVERSITY OF CALIFORNIA LOS ANGELES
- **Principal Investigator:** Steven John Jonas
- **Activity code:** DP5 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $390,000
- **Award type:** 5
- **Project period:** 2019-09-16 → 2025-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10693313

## Citation

> US National Institutes of Health, RePORTER application 10693313, Nanoscience-Inspired Acoustofluidic Assembly Lines for Gene and Cellular Therapies (5DP5OD028181-05). Retrieved via AI Analytics 2026-05-21 from https://api.ai-analytics.org/grant/nih/10693313. Licensed CC0.

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