# SCGE Comparative Studies Supplement

> **NIH NIH U01** · UNIVERSITY OF WASHINGTON · 2021 · $158,307

## Abstract

The goal of the U01 is to apply genome editors in organoid cultures to establish a predictive model for adverse
events in human kidney cell types, including both acute and chronic disorders with life-threatening consequences.
Genome editing platforms enable the efficient manipulation of specific DNA sequences in the human genome,
and therefore have enormous potential as therapeutics. The studies proposed in this supplemental application
bring together two complementary SCGE Consortium teams to advance combined in vitro and in vivo safety and
efficiency testing for somatic cell genome editing. The in vitro component addresses human cells in our 3D
organoid model, a rapid and high-throughput testing platform for preclinical assessments. However, the degree
to which organoids can predict responses in vivo remains unclear thus this system will be integrated with the
synergistic studies in the SCGE Testing Center to address topics of interest to regulatory agencies such as
dose response, editing delivery components, safety, and potential toxicities to guide preclinical/clinical
monitoring. As a prototype for proof-of-concept, studies focus on the kidney because ~20 million Americans
suffer from chronic kidney disease. Kidney disease is on the rise particularly in children, a population that suffers
disproportionately from genetic causes that could be targeted with genome editing. CRISPR/Cas9 editing
delivered by adeno-associated virus (AAV) is proposed to introduce specific edits at the AAVS1 target locus.
Collectively, these studies will establish a new regulatory paradigm which can be applied to a range of tissues
and diseases.

## Key facts

- **NIH application ID:** 10448959
- **Project number:** 3U01DK127553-03S1
- **Recipient organization:** UNIVERSITY OF WASHINGTON
- **Principal Investigator:** Benjamin Solomon Freedman
- **Activity code:** U01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $158,307
- **Award type:** 3
- **Project period:** 2021-09-01 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10448959

## Citation

> US National Institutes of Health, RePORTER application 10448959, SCGE Comparative Studies Supplement (3U01DK127553-03S1). Retrieved via AI Analytics 2026-05-29 from https://api.ai-analytics.org/grant/nih/10448959. Licensed CC0.

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