# Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes

> **NIH NIH U01** · DUKE UNIVERSITY · 2020 · $656,092

## Abstract

Abstract:
 Genome editing technologies have significant potential to treat a variety of devastating human diseases and
disorders. However, there are a number of challenges that genome editing therapies must overcome to reach
their full promise. Specifically, there are many possible adverse consequences that are unique to genome editing
tools, such as genome integrity, immune responses, and loss of therapeutic efficacy due to cell turnover, for
which there are currently are no optimal systems for rigorous assessment. Moreover, these consequences are
unique to human physiology, genome sequence, and immune systems, and therefore typical animal models are
not completely informative. To address this unmet need, we have assembled a team of collaborative
investigators that have developed advanced genome editing strategies and methods for engineering human
microphysiological tissue systems that recapitulate human physiology and function, with an emphasis on skeletal
and cardiac muscle. We will combine these technologies in this proposal to systemically evaluate tissue
physiology, genomic alterations, tissue regeneration, and immune response in response to various genome
editing strategies and delivery methods. Specifically, this will include comprehensive and unbiased mapping of
unintended modifications to human genome sequences, including at on-target and off-target sites. We will also
determine the role of resident tissue stem cells, cell turnover, and tissue injury and regeneration in the stability
of genome editing. Finally, we can incorporate immune cells into these microphysiological tissues to understand
the consequences of immunity to bacteria-derived genome editing components. Collectively, this proposal will
develop a platform to systematically address the most significant challenges to realizing the transformative
potential of genome editing therapies in human tissues.

## Key facts

- **NIH application ID:** 10001507
- **Project number:** 8U01HL156348-02
- **Recipient organization:** DUKE UNIVERSITY
- **Principal Investigator:** Nenad Bursac
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $656,092
- **Award type:** 8
- **Project period:** 2019-09-01 → 2023-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10001507

## Citation

> US National Institutes of Health, RePORTER application 10001507, Microphysiological Human Tissue Systems for Monitoring of Genome Editing Outcomes (8U01HL156348-02). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10001507. Licensed CC0.

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