Oligonucleotide-Based Therapy for FTD/ALS Caused by the C9orf72 Repeat Expansion: A Perspective
Figure 2
Chemical structures of commonly used oligonucleotides. Nucleic acids can bind to RNA targets that are complementary to their own sequence and trigger target degradation. However, unmodified DNAs/RNAs are subject to endonuclease degradation. Thus, to target RNAs, modifications to this primary structure are needed. Modifications to the nucleic acid backbone can lead to structures that can better interact with the target and are resistant to endonucleases. Modifications in the sugar ring are also possible and give rise to nucleic acids that mimic RNA, have better targeting, and are also resistant to endonuclease action.
