# In vivo production of a potassium channel inhibitor for immunomodulation

> **NIH NIH R21** · BAYLOR COLLEGE OF MEDICINE · 2020 · $198,125

## Abstract

PROJECT SUMMARY
 Approximately 50 million people suffer from one or more autoimmune diseases in the US alone. Over the
last decades, the discovery of biologics has considerably improved the care for patients with autoimmune
diseases. However, current treatments require weekly or more frequent injections and can lead to missed doses
and to poor patient compliance, especially in the younger population. Slow-release formulations of therapeutics
have been developed, but these still require multiple administrations. Since many of the novel therapeutics on
the market or being developed to treat multiple sclerosis, rheumatoid arthritis, and other T lymphocyte-mediated
chronic autoimmune diseases are biologics, our long-term goal is to have the body itself produce the therapeutic
for sustained release, using the liver as a “bio-factory”. As a first step, through this high risk / high reward R21
project, as a proof of concept, we propose to develop an adeno-associated virus (AAV) based methodology to
induce the production of an immunomodulatory peptide by rat liver and test its efficacy in rat models of multiple
sclerosis and rheumatoid arthritis. Specifically, we will define the relationships between vector dose and liver
transduction, as well as immunomodulator activity in the plasma (Specific Aim 1). We will then determine the
efficacy of this novel gene therapy for correcting two autoimmune diseases, multiple sclerosis and rheumatoid
arthritis, in rats (Specific Aim 2).

## Key facts

- **NIH application ID:** 9830010
- **Project number:** 5R21AI136876-02
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** Christine Beeton
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $198,125
- **Award type:** 5
- **Project period:** 2018-12-01 → 2021-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9830010

## Citation

> US National Institutes of Health, RePORTER application 9830010, In vivo production of a potassium channel inhibitor for immunomodulation (5R21AI136876-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/9830010. Licensed CC0.

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