Molecular BiologyNature CommunicationsPeer-reviewed
CRISPR/Cas9-Mediated Excision of ALS/FTD-Causing Hexanucleotide Repeat Expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro
Katharina E. Meijboom, Abbas Abdallah, Nicholas P. Fordham…·Christian Mueller
This study removes a hexanucleotide repeat expansion in C9ORF72 using CRISPR/Cas9 in cell and animal models, reducing disease hallmarks.