Gene supplementation in Vanishing White Matter mice ameliorates the disease
Anne E.J. Hillen, Robbert Zalm, Marjo S. van der Knaap, Vivi Majella Heine
This study shows that gene supplementation in astrocytes of Vanishing White Matter mice improves motor skills and CNS pathology without side effects.
bioRxiv · Dec 9, 2025 · PreprintNeuroscience