A recent article from SciTechDaily describes the promising approach instituted at a private, non-profit biomedical research firm in San Francisco that incorporates data science, AI, stem cell biology, CRISPR, and proteomics (the study of the proteins that that drive cellular activity) to search for disease cures. Gladstone Institutes also has strong ties to three nearby universities UC San Francisco, UC Berkeley, and Stanford University.
Gladstone Institutes has introduced a novel, “genetics-first” framework that systematically pairs specific vitamins with rare genetic conditions. Instead of evaluating a single disease and seeking out a custom cure, researchers used CRISPR gene editing to remove individual genes from human cells, testing which cell lines thrived when exposed to high doses of vitamins.
Through this method, they discovered that high-dose vitamin B3 (niacin) dramatically counteracts NAXD deficiency—a devastating and typically fatal childhood metabolic disorder. When tested on the first-ever mouse model of the disease, the vitamin B3 treatment successfully corrected cellular energy imbalances, eliminated brain inflammation, and extended survival by more than fortyfold. Because early intervention was critical to these results, researchers are advocating for NAXD deficiency to be added to newborn screening panels.
This scalable framework has already highlighted dozens of other genetic mutations that may respond to vitamin B2 or B3, offering a promising, cost-effective path toward treating hundreds of rare conditions using widely available micronutrients.
