# Gene Editing Core

> **NIH NIH U19** · JACKSON LABORATORY · 2024 · $404,630

## Abstract

PROJECT SUMMARY GENE EDITING CORE
The Liu laboratory at the Broad Institute of MIT and Harvard will serve as the Gene Editing Core for the Program.
Developing customized editing strategies that maximize therapeutic editing while minimizing undesired
byproducts is important for the success of all Research Projects. A centralized Gene Editing Core staffed by Liu
laboratory members will enhance the Program's efficiency by preventing bottlenecks in gene editing analyses
and ensuring consistency in methods and materials used in the proposed projects. The Gene Editing Core has
three central aims: 1) to assess on-target editing activity across all Research Projects from isolated cell lines and
from animal tissue samples; 2) to assess off-target editing activity in treated cells; 3) to optimize gene editing
technologies for therapeutic targets. The Core will play an essential role in generating rigorous analyses of gene
editing outcomes for the IND submission of our trailblazer project, coordinating closely with the Preclinical Mouse
Model Core analyze samples from large-scale efficacy studies. Additionally, the Core will facilitate the maturation
of the Follower Projects by continuing to develop the latest base editing and/or prime editing technologies in
model systems representative of each Follower Project's therapeutic target. Synergy between the Core and the
Follower Projects will enable the co-development of new genome editing tools that maximize on-target editing
efficiencies, minimize undesirable gene editing byproducts and off-target editing events, and maximize
compatibility with in vivo delivery methods of potential therapeutic relevance.

## Key facts

- **NIH application ID:** 10842405
- **Project number:** 5U19NS132304-02
- **Recipient organization:** JACKSON LABORATORY
- **Principal Investigator:** DAVID R LIU
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $404,630
- **Award type:** 5
- **Project period:** 2023-05-16 → 2028-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10842405

## Citation

> US National Institutes of Health, RePORTER application 10842405, Gene Editing Core (5U19NS132304-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10842405. Licensed CC0.

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