# Urgent Supplement: Correcting genetic disorders using predictable CRISPR/Cas9-induced exon skipping

> **NIH NIH R21** · BRIGHAM AND WOMEN'S HOSPITAL · 2020 · $139,773

## Abstract

Project Summary
The rapid spread of SARS-CoV2 presents an unprecedented challenge to urgently control disease
morbidity, mortality, and spread. Antiviral drugs including remdesivir, favipiravir, and EIDD-2801 have
emerged as front-line treatments. However, there are toxicity concerns for these drugs, especially as
they are most effective when given at high doses early in disease progression. The ability to administer
these antivirals more safely, particularly in less severely affected individuals, will allow for earlier
treatment. In this project, we combine experimental genomic approaches and genetic association
studies to understand and mitigate toxicity of SARS-CoV-2 antiviral drugs.
In Aim 1, we will use state-of-the-art genomic screening to identify human genes that mediate toxicity
of SARS-CoV-2 antiviral drugs in liver and intestinal cell lines. In Aim 2, we will examine genetic
associations of remdesivir efficacy and toxicity from ongoing clinical trials. In Aim 3, we will combine
this information to test approved pharmaceuticals or nutrients which are known to target or interact with
genes we identify to determine if any mitigate drug cytotoxicity. We will also determine whether there
are any common genetic or disease conditions for which antiviral dosing may be inadvisable.
Altogether, we aim to provide rapid and actionable insight on the toxicity of front-line SARS-CoV-2
antiviral drugs.

## Key facts

- **NIH application ID:** 10163567
- **Project number:** 3R21HG010391-02S1
- **Recipient organization:** BRIGHAM AND WOMEN'S HOSPITAL
- **Principal Investigator:** Christopher Cassa
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $139,773
- **Award type:** 3
- **Project period:** 2020-06-01 → 2021-01-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10163567

## Citation

> US National Institutes of Health, RePORTER application 10163567, Urgent Supplement: Correcting genetic disorders using predictable CRISPR/Cas9-induced exon skipping (3R21HG010391-02S1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10163567. Licensed CC0.

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