Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective

Figure 4

Strategy for ASO-based knocking down of the C9orf72 gene in a c9FTD/ALS patient. Using antisense oligonucleotides (ASO) targeting the GGGGCC repeat expansion can lead to selective knocking down of the mutant allele which could otherwise cause the formation of RNA foci. It also preserves the expression of the normal allele that may have an important function for cell survival. ASO is indicated by a blue line, dot line indicates introns, and squares indicate exons.