Nicotinamide Riboside Restores Mitochondrial Function in Alpers’ Syndrome: Preclinical Findings
Synopsis
Alpers' syndrome is a devastating early-onset neurodegenerative disorder caused by POLG mutations impairing mitochondrial DNA replication. Patient-derived iPSCs were used to create brain organoids and neural stem cells, which replicated the mitochondrial and neuronal defects seen in patients. Treatment with nicotinamide riboside (NR) improved mitochondrial function in these organoids, suggesting NR could be a potential therapeutic strategy for Alpers' syndrome and other mitochondrial disorders with similar mechanisms.
Journal
International Journal of Biological Sciences