Article: The Effects of NAD+ Boosting in Mitochondrial DNA Depletion Syndrome: Preclinical Findings

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The Effects of NAD+ Boosting in Mitochondrial DNA Depletion Syndrome: Preclinical Findings

Synopsis

Patients with mtDNA depletion syndrome 3 (MTDPS3) often die young from liver failure due to severely reduced mitochondrial DNA. Research into treatments has been difficult because MTDPS3 liver cells are hard to grow and study. This study created DGUOK-deficient liver-like cells from induced pluripotent stem cells (iPSCs) to model the disease and test drugs. Nicotinamide adenine dinucleotide (NAD+) improved mitochondrial function in these cells by activating PGC1α, a key regulator of energy production. NAD+ also increased ATP production in MTDPS3 rats and in cells lacking another related gene, RRM2B, indicating potential broader effectiveness. These findings show that patient-like cells can model MTDPS3 and help find treatments for mitochondrial DNA depletion disorders.

Journal

Cell Reports

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