RNA-Based Therapeutic Approaches
RNA-based therapeutics can be classified based on the mechanism of activity, and it include inhibitors of mRNA translation, the agents of RNA interference (RNAi), catalytically active RNA molecules, and RNAs that bind proteins and other molecularligands. RNA therapeutics have improvements in synthetic and natural nucleic acid carriers and the development of chemically modified oligonucleotides. RNA-based therapeutics currently under clinical investigation for diseases like genetic disorders, HIV infection, and various cancers. RNA is inherently unstable, mostly immunogenic, and requires a delivery vehicle for efficient transport to the targeted cells. RNA-based therapeutics that broaden the range of “drug-able” targets beyond the scope of existing pharmacological drugs.
- mRNA splicing and links to disease
- mRNA processing and human disease
- MDS, leukemia and splicing