# Chemical approaches for precision genome editing

> **NIH NIH R01** · BROAD INSTITUTE, INC. · 2021 · $74,047

## Abstract

PROJECT SUMMARY
CRISPR-Cas9 is an RNA-guided endonucleases that is being actively used for sequence-specific DNA
recognition, genome engineering, targeted transcriptional activation/repression and genome imaging. Cas9 is
being developed as a gene therapy agent for multiple pathologies, including HIV, vision disorders, muscular
dystrophy, and hereditary disorders. The precision control of specificity of CRISPR-Cas9 is required as off-
target effects and chromosomal translocations are observed at elevated activity. Further, the ease of targeting
catalytically impaired Cas9 to any genomic locus has resulted in transformative technologies. For example, the
fusion of catalytically inactive Cas9 (dCas9) to transcriptional activators or repressors has enabled gene
transcription and repression; fusion of catalytically impaired Cas9 to base-modifying enzymes has allowed base
conversion (e.g., C→T) at specific genomic sites; dCas9‒GFP fusion has made imaging genomic loci possible;
and dCas9‒acetyltransferases or deacetylases fusion has enabled epigenome editing. We propose to apply
chemical and genetic approaches develop reagents and methods that will allow precision control of specificity
of CRISPR-Cas9.
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## Key facts

- **NIH application ID:** 10389932
- **Project number:** 3R01GM137606-01A1S1
- **Recipient organization:** BROAD INSTITUTE, INC.
- **Principal Investigator:** Amit Choudhary
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2021
- **Award amount:** $74,047
- **Award type:** 3
- **Project period:** 2021-04-01 → 2025-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10389932

## Citation

> US National Institutes of Health, RePORTER application 10389932, Chemical approaches for precision genome editing (3R01GM137606-01A1S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10389932. Licensed CC0.

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