# SCGE Comparative Studies Supplement

> **NIH NIH U42** · UNIVERSITY OF CALIFORNIA AT DAVIS · 2021 · $327,913

## Abstract

The Nonhuman Primate Testing Center supports studies that advance the genome editing field, contributing to
the future translation of these technologies for the treatment of human disease. 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 a 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 preclinical primate model 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. This is an ideal candidate because it is expressed in human and rhesus with conserved
sequences. In vitro studies in human organoids will be paralleled with investigations that utilize a direct
ultrasound guided intrarenal approach in young monkeys. Collectively, these studies will compare human
kidney organoids with outcomes in nonhuman primates to establish a new regulatory paradigm which can be
applied to a range of tissues and diseases.

## Key facts

- **NIH application ID:** 10445645
- **Project number:** 3U42OD027094-03S1
- **Recipient organization:** UNIVERSITY OF CALIFORNIA AT DAVIS
- **Principal Investigator:** DENNIS J. HARTIGAN-O'CONNOR
- **Activity code:** U42 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $327,913
- **Award type:** 3
- **Project period:** 2019-04-15 → 2024-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10445645

## Citation

> US National Institutes of Health, RePORTER application 10445645, SCGE Comparative Studies Supplement (3U42OD027094-03S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10445645. Licensed CC0.

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