Authors: Cristin D. Davidson, Nafeeza F. Ali, Matthew C. Micsenyi, Gloria Stephney, Sophie Renault, Kostantin Dobrenis, Daniel S. Ory, Marie T. Vanier, Steven U. Walkley
DOI: 10.1371/journal.pone.0006951
Abstract Summary
A drug vehicle, 2-hydroxypropyl-β-cyclodextrin (CD), unexpectedly proved therapeutic for Niemann-Pick type C disease in mice. Regular CD treatment delayed disease onset, reduced harmful cholesterol and lipid buildup in neurons, and significantly extended lifespan in both NPC1 and NPC2 deficient mice—outperforming previous treatments. However, CD showed no benefit for related storage diseases GM1 gangliosidosis and MPS IIIA.
Why Brain? 🧠
Cyclodextrin treatment significantly extends lifespan and reduces harmful brain storage in mouse models of Niemann-Pick C disease, offering a promising new therapeutic approach for this fatal disorder.
The image is AI-generated for illustrative purposes only. Courtesy of Midjourney.
