disease course: 10 months at time of biopsy
drug treatment: No ASO Treatment - Cells were exposed to cytofectin transfection reagent for four hours, but were not transfected with an ASO
disease course: 6 years at time of biopsy
disease course: 2.5 years at time of biopsy
disease course: 2 years at time of biopsy
genotype: C9orf72 Intron 1 Hexanucleotide Expansion
disease course: 4 years at time of biopsy
drug treatment: Transfection Control - Cells were treated with a control ASO which does not have any human genomic target.
diagnosis: Sporadic ALS (C9orf72 expansion)
disease course: 1 year at time of biopsy
drug treatment: ASO Treatment - Cells were transfected with a nuclear RNase-H dependent antisense oligonucleotide targeting Exon 2 of Hs C9orf72. This ASO has been shown in previous work to efficiently knockdown all known isoforms of C9orf72.
disease course: 14 years at time of biopsy