Potential therapeutic applications of RNA cap analogs.

Abstract:

: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.

journal_name

Future Med Chem

authors

Ziemniak M,Strenkowska M,Kowalska J,Jemielity J

doi

10.4155/fmc.13.96

subject

Has Abstract

pub_date

2013-06-01 00:00:00

pages

1141-72

issue

10

eissn

1756-8919

issn

1756-8927

journal_volume

5

pub_type

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