Therapeutic suppression of proteolipid protein rescues Pelizaeus-Merzbacher Disease in mice
Matthew S. Elitt, Lilianne Barbar, H. Elizabeth Shick, Berit E. Powers, Yuka Maeno-Hikichi, Mayur Madhavan, Kevin Cameron Allan, Baraa S. Nawash, Artur S. Gevorgyan, Stevephen Hung, Zachary Scott Nevin, Hannah E. Olsen, Midori Hitomi, Daniela Schlatzer, Hien Tran Zhao, , Paul J. Tesar
This paper shows that reducing PLP1 expression in a mouse model of PMD improves myelination, motor function, and lifespan, supporting PLP1 suppression as a therapeutic strategy.
Nature · Jul 1, 2020Peer-reviewedNeuroscience