Nicotinamide Riboside Restores Exercise Capacity and Mitochondrial Function in Barth Syndrome: Preclinical Findings

Synopsis

Barth syndrome, caused by tafazzin mutation, leads to mitochondrial dysfunction and severe exercise intolerance. In a Drosophila model, NR supplementation restored exercise capacity and mitochondrial respiration via activation of sir2 and pgc-1α pathways. Overexpression of pgc-1α increased cardiolipin content, improving mitochondrial membrane composition. Muscle and neurons were identified as key targets for therapy. This study highlights NR's therapeutic potential in Barth syndrome.

Journal

Disease Models & Mechanisms