# iPSC Gene Editing Core

> **NIH NIH P01** · BOSTON UNIVERSITY MEDICAL CAMPUS · 2024 · $427,164

## Abstract

SUMMARY/ABSTRACT: CORE A (iPSC Gene Editing Core; iGEC)
The iPSC Gene Editing Core (iGEC) will be a state-of-the-art Core Laboratory at the Boston University
Chobanian & Avedisian School of Medicine that builds on the extensive experience of our investigators. The
iGEC will serve the aims of Projects 1, 2, 3, and 4 through the application of state-of-the-art technical approaches
to manipulating iPSC and iBC populations and generating lentiviral constructs used in each Project. The reagents
that will be generated will be central to the successful completion of the individual projects composing this
Program Project Grant (PPG), providing the ability to perform experiments in a significantly larger number of
genetic backgrounds than would otherwise be possible. Our three specific aims are as follows. Aim 1: To edit
existing iPSC and iBC lines derived from patients with genetic lung diseases to correct disease-associated
variants or introduce CRISPRi or CRISPRa knock-in constructs to facilitate genetically-controlled disease
modeling experiments featured in individual PPG projects. Aim 2: To perform rigorous QC testing and
biobanking of edited iPSC lines generated for and utilized in all PPG projects. Aim 3: To clone, package, titer,
bank, and distribute lentiviral constructs for use in PPG projects. PPG investigators have significant collective
expertise in a suite of technologies, including iPSC differentiation, gene editing of iPSCs and their derivatives,
and the application of lentiviruses to track cell populations, modulate gene expression, or read out pathway
activation, that will be applied in Projects 1, 2, 3, and 4 of this PPG. The iGEC will centralize and standardize
these innovative approaches and reagents to make them available for PPG projects. The iGEC will provide gene
editing services to generate edited versions, including the introduction or correction of variants as well as the
generation of knock-in constructs, of existing iPSC lines utilized in individual Projects. It will likewise clone new
lentiviral constructs to facilitate the targeted knockdown or overexpression of genes of interest using
CRISPRi/CRISPRa technology. It will apply rigorous QC protocols to document the provenance of edited iPSC
lines through DNA “fingerprinting”, confirm their genetic stability through karyotyping, and confirm their
pluripotency. Finally, it will serve as a central repository for all edited iPSCs and lentiviral constructs utilized in
the PPG, applying best practices developed over time to minimize iPSC passage number and maintain the bank.

## Key facts

- **NIH application ID:** 10768964
- **Project number:** 1P01HL170952-01
- **Recipient organization:** BOSTON UNIVERSITY MEDICAL CAMPUS
- **Principal Investigator:** ANDREW A WILSON
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $427,164
- **Award type:** 1
- **Project period:** 2024-03-01 → 2029-02-28

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10768964

## Citation

> US National Institutes of Health, RePORTER application 10768964, iPSC Gene Editing Core (1P01HL170952-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10768964. Licensed CC0.

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