GAPDH controls extracellular vesicle biogenesis and enhances therapeutic potential of EVs in silencing the Huntingtin gene in mice via siRNA delivery
Ghulam Hassan Dar, Cláudia C. Mendes, Wei‐Li Kuan, Alfina A. Speciale, Mariana Conceição, André Görgens, Inna Uliyakina, Miguel J. Lobo, Wooi Fang Lim, Samir EL Andaloussi, Imre Mäger, Thomas C. Roberts, Roger A. Barker, Deborah C. I. Goberdhan, CLIVE G. WILSON, Matthew J. A. Wood
This study shows GAPDH facilitates EV biogenesis and enables efficient siRNA delivery to the brain, reducing huntingtin gene expression in a mouse model.
Nature Communications · Nov 18, 2021Peer-reviewedNeuroscience