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Targeting RNA in mammalian systems with small molecules.

Publication ,  Journal Article
Donlic, A; Hargrove, AE
Published in: Wiley interdisciplinary reviews. RNA
July 2018

The recognition of RNA functions beyond canonical protein synthesis has challenged the central dogma of molecular biology. Indeed, RNA is now known to directly regulate many important cellular processes, including transcription, splicing, translation, and epigenetic modifications. The misregulation of these processes in disease has led to an appreciation of RNA as a therapeutic target. This potential was first recognized in bacteria and viruses, but discoveries of new RNA classes following the sequencing of the human genome have invigorated exploration of its disease-related functions in mammals. As stable structure formation is evolving as a hallmark of mammalian RNAs, the prospect of utilizing small molecules to specifically probe the function of RNA structural domains and their interactions is gaining increased recognition. To date, researchers have discovered bioactive small molecules that modulate phenotypes by binding to expanded repeats, microRNAs, G-quadruplex structures, and RNA splice sites in neurological disorders, cancers, and other diseases. The lessons learned from achieving these successes both call for additional studies and encourage exploration of the plethora of mammalian RNAs whose precise mechanisms of action remain to be elucidated. Efforts toward understanding fundamental principles of small molecule-RNA recognition combined with advances in methodology development should pave the way toward targeting emerging RNA classes such as long noncoding RNAs. Together, these endeavors can unlock the full potential of small molecule-based probing of RNA-regulated processes and enable us to discover new biology and underexplored avenues for therapeutic intervention in human disease. This article is categorized under: RNA Methods > RNA Analyses In Vitro and In Silico RNA Interactions with Proteins and Other Molecules > Small Molecule-RNA Interactions RNA in Disease and Development > RNA in Disease.

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Published In

Wiley interdisciplinary reviews. RNA

DOI

EISSN

1757-7012

ISSN

1757-7004

Publication Date

July 2018

Volume

9

Issue

4

Start / End Page

e1477

Related Subject Headings

  • Small Molecule Libraries
  • RNA Splice Sites
  • RNA Probes
  • RNA
  • Phenotype
  • Nervous System Diseases
  • Neoplasms
  • Humans
  • Animals
  • 3105 Genetics
 

Citation

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Donlic, A., & Hargrove, A. E. (2018). Targeting RNA in mammalian systems with small molecules. Wiley Interdisciplinary Reviews. RNA, 9(4), e1477. https://doi.org/10.1002/wrna.1477
Donlic, Anita, and Amanda E. Hargrove. “Targeting RNA in mammalian systems with small molecules.Wiley Interdisciplinary Reviews. RNA 9, no. 4 (July 2018): e1477. https://doi.org/10.1002/wrna.1477.
Donlic A, Hargrove AE. Targeting RNA in mammalian systems with small molecules. Wiley interdisciplinary reviews RNA. 2018 Jul;9(4):e1477.
Donlic, Anita, and Amanda E. Hargrove. “Targeting RNA in mammalian systems with small molecules.Wiley Interdisciplinary Reviews. RNA, vol. 9, no. 4, July 2018, p. e1477. Epmc, doi:10.1002/wrna.1477.
Donlic A, Hargrove AE. Targeting RNA in mammalian systems with small molecules. Wiley interdisciplinary reviews RNA. 2018 Jul;9(4):e1477.
Journal cover image

Published In

Wiley interdisciplinary reviews. RNA

DOI

EISSN

1757-7012

ISSN

1757-7004

Publication Date

July 2018

Volume

9

Issue

4

Start / End Page

e1477

Related Subject Headings

  • Small Molecule Libraries
  • RNA Splice Sites
  • RNA Probes
  • RNA
  • Phenotype
  • Nervous System Diseases
  • Neoplasms
  • Humans
  • Animals
  • 3105 Genetics