Antisense oligonucleotides and other genetic therapies made simple

Antisense oligonucleotides and other genetic therapies made simple.

Many genetic neurological diseases result from the dysfunction of single proteins. Genetic therapies aim to modify these disease-associated proteins by targeting the RNA and DNA precursors. This review provides a brief overview of the main types of genetic therapies, with a focus on antisense oligonucleotides (ASOs) and RNA interference (RNAi). We use examples of new genetic therapies for spina...

Antisense Oligonucleotides: Strategies and Applications

Designing Antisense Oligonucleotides

Antisense Oligonucleotides Targeting Protein

Designing Antisense Oligonucleotides

Antisense oligonucleotides have been used for a number of years to modify the expression of specific genes both in vivo and in vitro (Scanlon et al., 1995). The most potent mode of antisense activity is through RNase H–mediated degradation of RNA (Figure 1). The RNase H– endonuclease specifically cleaves RNA only when it is hybridized as a heteroduplex with DNA. In general, oligonucleotides tha...