RNA therapeutics include RNA molecules that encode proteins, RNA molecules that target nucleic acids (either DNA or RNA) and RNA molecules that target proteins. Those that target nucleic acids include single-stranded antisense oligonucleotides (ASOs) and double-stranded molecules known to operate through the RNA interference (RNAi) pathway. RNA therapeutics that target proteins are called RNA aptamers.
ASOs are short stretches or modified DNA of 13-25 nucleotides which act by preventing mRNA from being translated into protein through blocking start of translation or tagging mRNA for degradation. An ASO may alter mRNA splicing, a maturation process necessary for translation to occur. RNAi based therapies use double-stranded molecules of small interfering RNAs (siRNAs) of 21-23 nucleotides or microRNAs, which prevent protein translation through degradation of mRNA. Double stranded molecules used in RNAi based therapies are more difficult to gain entry into cells compared with single stranded ASOs.
RNA aptamers are designed to bind a specific site on a specific protein to affect its function. RNA aptamers have rapid action and reversibility.
Documentaries, videos and podcasts
- Cluster: RNA-based and RNA-targeted therapeuticsA cluster of topics related to RNA-based therapeutics and RNA-targeted therapeutics. RNA-based therapeutics include RNA and oligonucleotide therapeutics that target RNA and proteins. RNA-targeted therapeutics include RNA and oligonucleotide therapeutics as well as small molecule drugs that target RNA.