Potential therapeutic applications of RNA cap analogs

Autor: Malwina Strenkowska, Jacek Jemielity, Joanna Kowalska, Marcin Ziemniak
Rok vydání: 2013
Předmět:
Zdroj: Future Medicinal Chemistry. 5:1141-1172
ISSN: 1756-8927
1756-8919
DOI: 10.4155/fmc.13.96
Popis: Cap analogs are chemically modified derivatives of the unique cap structure present at the 5´ end of all eukaryotic mRNAs and several non-coding RNAs. Until recently, cap analogs have served primarily as tools in the study of RNA metabolism. Continuing advances in our understanding of cap biological functions (including RNA stabilization, pre-mRNA splicing, initiation of mRNA translation, as well as cellular transport of mRNAs and snRNAs) and the consequences of the disruption of these processes – resulting in serious medical disorders – have opened new possibilities for pharmaceutical applications of these compounds. In this review, the medicinal potential of cap analogs in areas, such as cancer treatment (including eIF4E targeting and mRNA-based immunotherapy), spinal muscular atrophy treatment, antiviral therapy and the improvement of the localization of nucleus-targeting drugs, are highlighted. Advances achieved to date, challenges, plausible solutions and prospects for the future development of cap analog-based drug design are described.
Databáze: OpenAIRE