NeuroscienceDisease Models & MechanismsPeer-reviewed
Inducible expression of human C9ORF72 36x G4C2 hexanucleotide repeats is sufficient to cause RAN translation and rapid muscular atrophy in mice
Fréderike W. Riemslagh, Esmay C. van der Toorn, Rob F. M. Verhagen…·Rob Willemsen
This paper introduces a new inducible mouse model expressing C9ORF72 G4C2 repeats that causes RAN translation and muscular atrophy, showing that early intervention can prevent but not reverse the phenotype.
17 citations