# Novel Therapeutics for Long QT Syndrome

> **NIH NIH R56** · COLUMBIA UNIVERSITY HEALTH SCIENCES · 2022 · $245,540

## Abstract

Project Summary:
Abnormal ion channel function in heart muscle cells induces cardiac arrhythmias such as long QT syndrome.
In preliminary experiments using pharmaceutical approach, we found that activation of Sigma 1 receptor could
alleviate the cellular phenotypes in human induced pluripotent stem cell (iPSC) and mouse models of the
genetic cardiac arrhythmias. The goal of this study is to examine the molecular mechanism underlying the
beneficial effect of Sigma receptor 1 activation on the phenotypes in cardiac ion channel regulation, action
potentials, contraction, mitochondrial function and gene expression in the genetic arrhythmia models. We will
use human iPSC and rodent models to accomplish our goal. In addition, we will design and develop new
Sigma 1 receptor agonists that are more suitable for cardiac arrhythmias, taking advantage of our expertise in
medicinal chemistry. Therefore, our translational study will provide new opportunity of drug development for the
genetic syndromic disorders.

## Key facts

- **NIH application ID:** 10705357
- **Project number:** 1R56HL158975-01A1
- **Recipient organization:** COLUMBIA UNIVERSITY HEALTH SCIENCES
- **Principal Investigator:** Masayuki Yazawa
- **Activity code:** R56 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $245,540
- **Award type:** 1
- **Project period:** 2022-09-22 → 2023-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10705357

## Citation

> US National Institutes of Health, RePORTER application 10705357, Novel Therapeutics for Long QT Syndrome (1R56HL158975-01A1). Retrieved via AI Analytics 2026-05-23 from https://api.ai-analytics.org/grant/nih/10705357. Licensed CC0.

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