Catalyst Awardee

Project Description

Restoring mitochondrial function using viral gene reconstitution therapy in aged muscle and heart

Olga Bielska, PhD; Eric Verdin, MD | Buck Institute for Research on Aging
Competition Sponsor: US National Academy of Medicine
Awardee Year: 2023

Mitochondrial dysfunction is a major contributing factor in age-related loss of skeletal muscle mass and cardiovascular disease. Mitochondrial division is a critical process for maintaining mitochondrial function.
We and others have recently discovered that loss of mitochondrial division protein (MDP) is an age-related phenomenon. It affects multiple tissues and is regulated by a complex molecular mechanism. In humans and in mice, the loss of MDP due to genetic mutations, results in cardiac and neuromuscular diseases, and severely shortened lifespan. We hypothesize that restoration of normal levels of MDP should help to revitalize mitochondrial function in aging and reverse negative age-related changes.
As a proof of a concept study we aim to test whether our adeno-associated virus (AAV) gene therapy can restore mitochondrial function and improve overall health and lifespan, first in mouse model of MDP deficiency, and then in aged mice. We will target heart and muscle tissues, which suffer the most from age-related mitochondrial division loss. If effective, this therapy could be extended to other affected organs.
Our AAV-based therapy has several unique advantages over traditional pharmaceutical interventions, including its ability to target specific tissues and cells, minimal side effects, and long-term benefits. Our research could significantly impact the field of healthy longevity, and we are committed to developing this therapy further. The Healthy Longevity Catalyst Award would provide invaluable support for our ongoing research. Overall, these advances hold great promise for the future of medicine and could lead to the development of cures for many age-related diseases.

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