Heart-on-Chip for Drug Efficacy Testing for Mitochondrial Diseases
Abstract
This project addresses the "Mitochondrial Disease" topic area. Mitochondria are structures within a cell that supply its energy. Mutations and deletions in mitochondrial DNA (mtDNA) are associated with a wide range of human diseases. There are no current cures for mitochondrial diseases, and current treatments only improve symptoms. Unfortunately, progress toward developing therapies for mitochondrial diseases has been impaired, as no effective benchtop models exist for the development and testing of drugs. This project seeks to develop a novel heart-chip platform that mimics the beating of the heart and its associated chemical and electrical signaling in a portable, miniaturized, benchtop format. This heart-chip will be engineered using patient-derived cells from both healthy donors and those that have a mitochondrial disease. Specifically, cells from patients with Kearns Sears Syndrome (KSS), Leigh’s Syndrome (LS), and mitochondrial myopathy, encephalopathy, lactic acidosis, and stroke (MELAS), three different mitochondrial diseases that affect the heart, will be utilized. These normal and diseased heart chips will then be used to test the safety and effectiveness of a novel drug, NV188, to improve the impaired heart function in these three mitochondrial diseases. Overall, successful completion of this project will result in the development of a novel test platform for developing therapeutics for military personnel and their dependents who suffer from mitochondrial diseases. In the future, the technology of this heart-chip platform can easily be extended to other organs such as the brain, blood-brain barrier, spinal cord, or the eye, as well as for the development of therapeutics for other diseases that affect these organs.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Mar 10, 2021
- Source ID
- W81XWH2010144
Entities
People
- Kartik Balachandran
Organizations
- United States Army
- University of Arkansas