Molecular BiologyScience Translational MedicinePeer-reviewed
A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export suppresses neurodegeneration
Lydia M. Castelli, Ya-Hui Lin, Álvaro Sánchez-Martínez…·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.
16 citations