# Genome Engineering & iPSC Center Core

> **NIH NIH P01** · WASHINGTON UNIVERSITY · 2020 · $156,125

## Abstract

Project Summary
Genome engineering and patient-derived pluripotent stem cell technology have dramatically changed our
abilities to understand disease pathways. These two technologies are recent additions to our investigative
armamentarium but have rapidly permeated all aspects of biomedical science, providing unprecedented power
to discover how perturbations of components in specific pathways lead to disease. The Genome Engineering
Core C is composed of 11 investigators and all of the necessary equipment, reagents and expertise required
to perform the experiments needed to support the research goals of the 3 projects outlined in this PO1 renewal
application entitled “New therapies for liver fibrosis and hyperproliferation in alpha1-AT deficiency
(ATD)”. Our facility is housed within the Washington University Genome Engineering and IPSC Center (GEiC)
and was established and is overseen by the Department of Genetics. It was created to facilitate the
implementation of these powerful new technologies in laboratories at Washington University. All projects
proposed within this PO1 renewal application plan to extensively utilize the services provided by the Core.
These experiments are largely aimed at the ongoing evaluation of modifying variants in ATD and their potential
exploitation for development of new treatments for this disorder. The services to be utilized include the design,
construction and validation of genome editing reagents (e.g. gRNAs, donor plasmids, and Cas9 derivatives).
The Core also produces modified cell lines using genome editing technologies, including gene knockout,
variant introduction, epitope tagging or gene replacement. These cell lines will be used to better understand
the biology of ATD and to assess the impact of variants selected for their potential to modify disease
progression and facilitate drug development. The Core will also design and generate materials needed for
rapid production of animal models harboring selected variants. The Core produces iPSCs from both skin
biopsies and the renal tubular epithelial cells present in urine samples. Genome engineering of iPSCs is now
routinely performed in the Core to introduce new variants and to convert disease-associated mutant alleles
back to wildtype or use as controls. Skin biopsies (fibroblasts) or urine (renal tubular epithelia) will be procured
from ATD patients or controls and the cells will be reprogrammed to produce iPSC lines. Genetically modified
patient-derived iPSCs will be generated using genome editing techniques. In addition, the personnel in the
Core provide assistance for investigators in performing genome engineering, particularly in the development of
new techniques and reagents, and for the maintenance and differentiation of iPSC lines.

## Key facts

- **NIH application ID:** 9994908
- **Project number:** 5P01DK096990-07
- **Recipient organization:** WASHINGTON UNIVERSITY
- **Principal Investigator:** JEFFREY D MILBRANDT
- **Activity code:** P01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $156,125
- **Award type:** 5
- **Project period:** — → —

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9994908

## Citation

> US National Institutes of Health, RePORTER application 9994908, Genome Engineering & iPSC Center Core (5P01DK096990-07). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/9994908. Licensed CC0.

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