RNA therapeutics: updates and future potential

Zhang, Caroline; Zhang, Biliang

doi:10.1007/s11427-022-2171-2

Cited by 58 publications

(28 citation statements)

References 108 publications

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“…RNA therapeutics progressed with the development of RIBOTAC and RiboSNAP moieties that can potentially degrade the target RNA. ,,, In 2020, RIBOTACs underwent their first preclinical evaluation . With the ability to modulate a wide range of cellular processes, RNA-targeted drug discovery possesses much more potential, and constant efforts are required to uncover the less explored possibilities of this field …”

Section: Discussionmentioning

confidence: 99%

RNA–Small-Molecule Interaction: Challenging the “Undruggable” Tag

Kaur,

Sharma,

Mundlia

et al. 2024

J. Med. Chem.

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RNA targeting, specifically with small molecules, is a relatively new and rapidly emerging avenue with the promise to expand the target space in the drug discovery field. From being "disregarded" as an "undruggable" messenger molecule to FDA approval of an RNA-targeting small-molecule drug Risdiplam, a radical change in perspective toward RNA has been observed in the past decade. RNAs serve important regulatory functions beyond canonical protein synthesis, and their dysregulation has been reported in many diseases. A deeper understanding of RNA biology reveals that RNA molecules can adopt a variety of structures, carrying defined binding pockets that can accommodate smallmolecule drugs. Due to its functional diversity and structural complexity, RNA can be perceived as a prospective target for therapeutic intervention. This perspective highlights the proof of concept of RNA−small-molecule interactions, exemplified by targeting of various transcripts with functional modulators. The advent of RNA-oriented knowledge would help expedite drug discovery. ■ SIGNIFICANCE• This Perspective highlights the importance of RNA as a drug target to solve the issues such as lack of therapeutic treatments for several diseases, increasing resistance against conventional drugs, and tackling the undruggability of various protein targets. • In-depth analysis of various aspects, i.e., small-moleculebinding modes, and current trends required to dig deep into this area of drug discovery are discussed. • The research discussed herein would aid in the development of small-molecule therapeutics.

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Section: Discussionmentioning

confidence: 99%

RNA–Small-Molecule Interaction: Challenging the “Undruggable” Tag

Kaur,

Sharma,

Mundlia

et al. 2024

J. Med. Chem.

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“…RNA therapy is a rapidly evolving class of drugs and therapies, which is transitioning from basic to clinical practice at unprecedented speed ( 72 ). CircRNA-based therapeutic approaches have promising medical and research applications in therapeutic drugs and protein replacement therapies for prophylactic vaccines ( 73 ).…”

Section: Discussionmentioning

confidence: 99%

Emerging roles of circular RNAs in cancer therapy-induced cardiotoxicity

Cheng

Qin

et al. 2023

Front. Cardiovasc. Med.

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Cancer therapy-induced cardiotoxicity (CTIC) is an important cause of death in cancer survivors which often results in the withdrawal or discontinuation of drugs. The underlying mechanisms of CTIC remain unclear. Circular RNAs (circRNAs) are a class of non-coding regulatory RNA molecules which have emerged in recent years. They are generated by back splicing and have powerful biological functions, including transcription and splicing, isolating or building macromolecular scaffolds to interfere with microRNA activity and signaling pathways, and acting as templates for translation. Moreover, circRNAs demonstrate high abundance and significant stability. CircRNAs can be used as novel biomarkers because they often function in a cell-type and tissue-specific manner. CircRNAs have attracted increasing attention in cardiovascular disease research, and recent studies exploring the role of circRNAs in CTIC have had promising results. This review will summarize the current understanding of circRNAs’ biogenesis, regulation and function. Their clinical potential as biomarkers, therapeutic agents and drug targets will also be explored.

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“…For this purpose, we also described a pancancer circRNA translation profile to mine the hidden proteome from circRNA based on our algorithm CICADA. These proteins may play carcinogenic or tumor suppressor roles or may become neoantigens, which will help discover, optimize, and develop new tools based on biological proteins for the early diagnosis and development of anticancer drugs 40 .…”

Section: Discussionmentioning

confidence: 99%

CICADA: a circRNA effort to Ghost proteome

Sun,

Fan,

Zhou

et al. 2023

Preprint

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Coding genes constitute only a small portion of the human genome. Recently, circular RNAs (circRNAs), commonly thought to be noncoding, have been demonstrated to be translated into novel proteins, which might mediate diverse biological processes. However, it remains challenging to identify translatable circRNAs and circRNA-coded proteins. Here, we introduce CICADA, an algorithm capable of evaluating the coding ability of circRNAs and inferring circRNA-coded products. We find that CICADA's performance is highly efficient and cost-effective compared to existing algorithms. Moreover, we used CICADA to successfully identify functional translatable circRNAs in esophageal squamous cell carcinoma and construct circRNA translation profiles across various cancers. CICADA provides an efficient tool to explore the hidden proteome encoded by translatable circRNAs.

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RNA therapeutics: updates and future potential

Cited by 58 publications

References 108 publications

RNA–Small-Molecule Interaction: Challenging the “Undruggable” Tag

RNA–Small-Molecule Interaction: Challenging the “Undruggable” Tag

Emerging roles of circular RNAs in cancer therapy-induced cardiotoxicity

CICADA: a circRNA effort to Ghost proteome

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