# Development of a microfluidic primary cell editing platform (pCEP) for personal gene therapy

> **NIH NIH R21** · JOHNS HOPKINS UNIVERSITY · 2021 · $184,852

## Abstract

Project Summary/Abstract (30 lines)
 Variation in treatment response remains a formidable obstacle when selecting ideal therapies for individual
patients suffering from various maladies. This problem, in particular, has led to the development of numerous
personalized medicine approaches aimed at tailoring therapy on a patient-by-patient basis in order to improve
outcomes. Many of these approaches require systematic and quantitative assays to be performed directly on
primary cells derived from each patient. However, this poses a particular challenge for translation into the
clinic, as the collection, testing, and manipulation of these cells are typically extremely expensive, time-
consuming and labor-intensive processes.
 Rapid improvements in genomic engineering methods have bolstered optimism for the prospect of
personalized gene therapy because these methods possess superior modularity, specificity, and capability for
rapid correction of disease-conferring genes. However, conventional gene delivery methods continue to require
laborious and cumbersome leukapheresis and target-cell purification procedures of patients' blood prior to
gene delivery. Moreover, gene engineering suffers from notable shortcomings, such as impermanent inhibition
of target functions and unpredictable off-target effects, and demands gene delivery techniques capable of
routine and repeated assays on target cells. Efficient multigene delivery methods are thus desirable to reduce
off-target toxicity by co-expressing therapeutic and protective markers in therapeutic cells.
 This project aims to construct a microfluidic primary cell editing platform (pCEP) for robust, affordable,
scalable and direct genetic modification of cells purified from bodily fluids. pCEP will selectively trap primary
target cells from blood via novel microscale vortices and efficiently co-deliver therapeutic genes and gene
editing machinery via automated electroporation of the captured cells. Unlike virus-mediated delivery, pCEP
employs a physical gene injection mechanism that offers lower operational costs and higher payloads with the
ability to directly deliver Good Manufacturing Practice (GMP)-grade genetic materials. Multiple genes of
interest can be sequentially injected into the cytosol in a dose-controlled manner by automated switching of
delivery solutions. The versatility and feasibility of the pCEP approach for clinical applications will be validated
by performing gene insertion and deletion using model systems for the immortalization of non-proliferating
somatic cells via hTERT plasmid injection and production of PD-1 knockout T-lymphocytes via CRISPR-cas9
gene editing, respectively. We envision that pCEP will provide an automated solution for genomic editing of
target primary cells directly from bodily fluids as well as a simple and facile means to assess unforeseen
adverse effects of newly developed gene-editing techniques for human cells.

## Key facts

- **NIH application ID:** 10105307
- **Project number:** 5R21CA229024-02
- **Recipient organization:** JOHNS HOPKINS UNIVERSITY
- **Principal Investigator:** Soojung Claire Hur
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $184,852
- **Award type:** 5
- **Project period:** 2020-02-12 → 2023-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10105307

## Citation

> US National Institutes of Health, RePORTER application 10105307, Development of a microfluidic primary cell editing platform (pCEP) for personal gene therapy (5R21CA229024-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10105307. Licensed CC0.

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