# Center for Genome Editing and Recording

> **NIH NIH RM1** · UNIVERSITY OF CALIFORNIA BERKELEY · 2020 · $2,074,678

## Abstract

CENTER FOR GENOME EDITING AND RECORDING: PROJECT SUMMARY
The ability to understand normal and pathologic functions of the human genome and to translate that
knowledge into effective therapies depends critically on determining how encoded genetic information confers
phenotype. Recent advances in DNA sequencing and bioinformatics have provided vast quantities of genomic
data that, in principle, hold the keys to advances in preventive medicine and therapeutic intervention. However,
realizing the promise of personalized medicine will require accurate interrogation and manipulation of DNA
sequences in situ at a scale and level of accuracy not currently available. The Center for Genome Editing and
Recording (CGER) will address these challenges by creating technologies to detect, alter and record the
sequence and output of the genome in individual cells and tissues. Building on the CRISPR-Cas9 genome
engineering technology harnessed from bacteria, CGER will couple the RNA-guided DNA cleavage activity of
the Cas9 enzyme to strategies for enhancing DNA sequence replacement using homology-directed double-
strand break repair. In parallel, CGER will conjugate Cas9 to DNA “base editing” domains to enable accurate
introduction or correction of point mutations without double-stranded DNA cleavage. Using cell-based assays,
CGER researchers will interrogate specific disease-associated loci in human cells to provide new biological
insights and uncover new therapeutic targets. Together, these approaches will enable the creation of any
desired sequence alteration at any locus with high specificity and efficiency, with profound implications for both
genome science and practical therapeutic intervention. To complement this suite of genome-manipulation
technologies, CGER will also develop a high-throughput pipeline for testing the functional gene expression
impacts of sequence variants responsible for human disease. This pipeline will identify and illuminate the
relationships between human genome sequence variations, target gene expression and interactions with other
genes. Finally, CGER will create new methods for permanently recording cell state changes in DNA so that
they can be read out in a single-cell RNA-seq format. This development of molecular cell recorders will focus
primarily on an evolving lineage tracer that, by enabling the generation of fate maps at unprecedented
resolutions, holds the promise to revolutionize studies of normal development and disease progression. To
achieve its goals, the Center brings together investigators with strengths in functional genomics, biochemistry,
chemical biology and medicine, all of whom have been instrumental in developing genome engineering tools
across multiple systems. The work proposed builds on capabilities at the University of California, the Innovative
Genomics Initiative, Harvard University and Massachusetts General Hospital to create transformative
capabilities and to access state-of-the-art research facilitie...

## Key facts

- **NIH application ID:** 9966002
- **Project number:** 5RM1HG009490-04
- **Recipient organization:** UNIVERSITY OF CALIFORNIA BERKELEY
- **Principal Investigator:** JENNIFER A DOUDNA
- **Activity code:** RM1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $2,074,678
- **Award type:** 5
- **Project period:** 2017-08-08 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9966002

## Citation

> US National Institutes of Health, RePORTER application 9966002, Center for Genome Editing and Recording (5RM1HG009490-04). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9966002. Licensed CC0.

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