A new study has uncovered a surprisingly simple way to treat a deadly newborn disease and dozens of other inherited conditions—by giving patients a boost of vitamin B2 or B3. Researchers used genome‑wide CRISPR screens to pinpoint genes that make cells vulnerable when these vitamins are missing. One gene, NAXD, was linked to a severe neurodevelopmental disorder that usually kills infants within months. By creating a mouse model that mimics the human disease, scientists discovered that the lack of NAXD causes toxic “mis‑metabolites” to pile up, draining the active form of vitamin B3 (NAD) from the brain and skin. This shortfall cripples essential metabolic pathways, especially those needed for brain development. Remarkably, feeding the mice a high‑dose vitamin B3 diet rescued them, extending their lives by more than 40‑fold, while a vitamin‑deficient diet accelerated death. The same vitamin‑based rescue was hinted at in early clinical case reports, suggesting an immediate, inexpensive therapy could be on the horizon. Beyond NAXD, the researchers identified dozens of other genetic diseases that respond to vitamin B2 or B3, reviving interest in vitamin biology as a fast‑track route to affordable treatments for rare disorders.
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