# Resource Core I: Genomic Outcomes Core

> **NIH NIH U19** · UNIVERSITY OF CALIFORNIA BERKELEY · 2024 · $714,766

## Abstract

PROJECT SUMMARY
The mission of “Resource Core I: the Genomic Outcomes Core” is to provide a suite of high-quality turn-key
DNA-based assays for monitoring the safety and efficacy of genome editing to support the Correction of
Neurological Disease via Allele Specific Excision of Pathogenic Repeats (CASEPR) program. The Genomic
Outcomes Core will provide the fundamental role of directly assessing the genomic effects of nuclease-based
therapeutic treatment at the target site (including measuring excision efficiency) and off-target regions of the
genome, both in cell culture models, and in experimental animal models; as such, the outcomes assessed by
the Core will complement the in vivo and biological impacts assessed by Cores II and III, respectively. For each
assay the Core will work with the appropriate project team or Core to onboard project-specific reagents or tissues
(RNPs, AAVs, cell lines, genomic DNAs etc). Working with the Administrative Core, results from analysis of all
the samples will be cataloged within the laboratory information management system (LIMS) and result reports
will be provided to the Project Teams via a standardized LIMS interface. These data will enable the selection of
preclinical leads, and provide critical feedback on genomic outcomes in cell and animal model systems. Further,
data on potency and specificity of genomic outcomes driven by preclinical Cas9-gRNA combinations for all three
Research Projects will provide a key basis for study reports to be provided to the FDA as part of regulatory filings
to ensure timely progress to IND. From a logistics perspective the consolidation of expertise for genomic assays
within the Genomic Outcomes Core will promote rapid and robust input-to-answer solutions for Project Teams
across the entire lifespan of all three Research Projects.

## Key facts

- **NIH application ID:** 10840849
- **Project number:** 5U19NS132303-02
- **Recipient organization:** UNIVERSITY OF CALIFORNIA BERKELEY
- **Principal Investigator:** FYODOR D URNOV
- **Activity code:** U19 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $714,766
- **Award type:** 5
- **Project period:** 2023-05-15 → 2028-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10840849

## Citation

> US National Institutes of Health, RePORTER application 10840849, Resource Core I: Genomic Outcomes Core (5U19NS132303-02). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10840849. Licensed CC0.

---

*[NIH grants dataset](/datasets/nih-grants) · CC0 1.0*