A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export suppresses neurodegeneration
Lydia M. Castelli, Ya-Hui Lin, Álvaro Sánchez-Martínez, Aytaç Gül, Kamallia Mohd Imran, Adrian Higginbottom, Santosh Kumar Upadhyay, Nóra M. Márkus, Raquel Rua Martins, Johnathan Cooper‐Knock, Claire Montmasson, Rebecca N. Cohen, Amy Walton, Claudia S. Bauer, Kurt J. De Vos, , Guillaume M. Hautbergue
This paper develops a cell-penetrant peptide that blocks C9ORF72-repeat RNA nuclear export, reducing neurodegeneration in preclinical models of ALS and FTD.
Science Translational Medicine · Mar 1, 2023Peer-reviewedMolecular Biology