Knocking out C9ORF72 exacerbates axonal trafficking defects associated with hexanucleotide repeat expansion and reduces levels of heat shock proteins II. Knocking out C9ORF72 exacerbates axonal trafficking defects associated with hexanucleotide repeat expansion and reduces levels of heat shock proteins II

wild type iPSCs were edited using CRISPR/Cas9 to knockout C9ORF72 followed by differentiation into neurons. Overall design: We assessed the contribution of C9ORF72 to MN degeneration by knocking out the start codon and comparing the transcriptome.