Current Advances in RNA Therapeutics for Human Diseases DOI Open Access
Hannah Zogg, Rajan Singh, Seungil Ro

et al.

International Journal of Molecular Sciences, Journal Year: 2022, Volume and Issue: 23(5), P. 2736 - 2736

Published: March 1, 2022

Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as genetic code containing necessary information for proper cell functioning. In years following these discoveries, vast knowledge seemingly endless roles have become better understood. Additionally, many new types RNAs discovered that seemed to no coding properties (non-coding RNAs), such microRNAs (miRNAs). The created a avenue treating various human diseases. However, is relatively unstable degraded fairly rapidly once administered; this has led development novel delivery mechanisms, nanoparticles increase stability well prevent off-target effects molecules. Current advances RNA-based therapies substantial promise preventing diseases disorders through fixing pathology instead merely symptomology similarly traditional therapeutics. Although therapeutics made it clinical trials, only few been FDA approved thus far. results trials ambivalent date, with some studies demonstrating potent efficacy, whereas others limited effectiveness and/or toxicity. Momentum building clinic therapeutics; future care will likely comprise promising This review focuses on current addresses challenges their development.

Language: Английский

Small molecule recognition of disease-relevant RNA structures DOI Creative Commons
Samantha M. Meyer,

Christopher C. Williams,

Yoshihiro Akahori

et al.

Chemical Society Reviews, Journal Year: 2020, Volume and Issue: 49(19), P. 7167 - 7199

Published: Jan. 1, 2020

Targeting RNAs with small molecules, a new frontier in drug discovery and development.

Language: Английский

Citations

141

Probing the dynamic RNA structurome and its functions DOI Creative Commons

Robert C. Spitale,

Danny Incarnato

Nature Reviews Genetics, Journal Year: 2022, Volume and Issue: 24(3), P. 178 - 196

Published: Nov. 8, 2022

RNA is a key regulator of almost every cellular process, and the structures adopted by molecules are thought to be central their functions. The recent fast-paced evolution high-throughput sequencing-based structure mapping methods has enabled rapid in vivo structural interrogation entire transcriptomes. Collectively, these studies shedding new light on long underestimated complexity organization transcriptome — structurome. Moreover, analyses challenging view that structurome static entity revealing how establish intricate networks alternative intramolecular intermolecular interactions ensembles dynamically regulated finely tune functions living cells. This understanding can shape cell phenotypes important implications for development RNA-targeted therapeutic strategies. In this Review, Spitale Incarnato discuss application transcriptomes cells providing insight into structurome, dynamics regulates processes.

Language: Английский

Citations

134

Long Noncoding RNAs in Plants DOI Open Access
Andrzej Wierzbicki, Todd Blevins, Szymon Świeżewski

et al.

Annual Review of Plant Biology, Journal Year: 2021, Volume and Issue: 72(1), P. 245 - 271

Published: March 23, 2021

Plants have an extraordinary diversity of transcription machineries, including five nuclear DNA-dependent RNA polymerases. Four these enzymes are dedicated to the production long noncoding RNAs (lncRNAs), which ribonucleic acids with functions independent their protein-coding potential. lncRNAs display a broad range lengths and structures, but they distinct from small guides interference (RNAi) pathways. frequently serve as structural, catalytic, or regulatory molecules for gene expression. They can affect all elements genes, promoters, untranslated regions, exons, introns, terminators, controlling expression at various levels, modifying chromatin accessibility, transcription, splicing, translation. Certain protect genome integrity, while others respond environmental cues like temperature, drought, nutrients, pathogens. In this review, we explain challenge defining lncRNAs, introduce machineries responsible production, organize knowledge by viewing throughout structure typical plant gene.

Language: Английский

Citations

131

The translatome of neuronal cell bodies, dendrites, and axons DOI Creative Commons
Caspar Glock, Anne Biever, Georgi Tushev

et al.

Proceedings of the National Academy of Sciences, Journal Year: 2021, Volume and Issue: 118(43)

Published: Oct. 20, 2021

Significance Proteins are the key drivers of neuronal synaptic function. The regulation gene expression is important for formation and modification synapses throughout lifespan. complexity dendrites axons imposes unique challenges protein supply at remote locations. discovery messenger RNAs (mRNAs) ribosomes near has shown that local synthesis represents an solution to this challenge. Here we used RNA sequencing ribosome determine directly population mRNAs present in process translation cell bodies, dendrites, axons. Thousands transcripts were differentially translated between body regions with over 800 exhibiting more dendritic–axonal compartment.

Language: Английский

Citations

129

Current Advances in RNA Therapeutics for Human Diseases DOI Open Access
Hannah Zogg, Rajan Singh, Seungil Ro

et al.

International Journal of Molecular Sciences, Journal Year: 2022, Volume and Issue: 23(5), P. 2736 - 2736

Published: March 1, 2022

Following the discovery of nucleic acids by Friedrich Miescher in 1868, DNA and RNA were recognized as genetic code containing necessary information for proper cell functioning. In years following these discoveries, vast knowledge seemingly endless roles have become better understood. Additionally, many new types RNAs discovered that seemed to no coding properties (non-coding RNAs), such microRNAs (miRNAs). The created a avenue treating various human diseases. However, is relatively unstable degraded fairly rapidly once administered; this has led development novel delivery mechanisms, nanoparticles increase stability well prevent off-target effects molecules. Current advances RNA-based therapies substantial promise preventing diseases disorders through fixing pathology instead merely symptomology similarly traditional therapeutics. Although therapeutics made it clinical trials, only few been FDA approved thus far. results trials ambivalent date, with some studies demonstrating potent efficacy, whereas others limited effectiveness and/or toxicity. Momentum building clinic therapeutics; future care will likely comprise promising This review focuses on current addresses challenges their development.

Language: Английский

Citations

128