Publication | Open Access

CRISPR deletion of the C9ORF72 promoter in ALS/FTD patient motor neurons abolishes production of dipeptide repeat proteins and rescues neurodegeneration

Neurodegenerative DiseasesCrispr DeletionAmyotrophic Lateral SclerosisDegenerative PathologyGene EditingNeurologyNeuroscienceNeurodegenerationGenome EditingC9orf72 PromoterMedicineCrisprDipeptide Repeat Proteins