Ribozymes ☆

Skilandat, Miriam; Zelger-Paulus, Susann; Sigel, Roland K. O.

doi:10.1016/b978-0-12-809633-8.07077-1

Cited by 2 publications

(2 citation statements)

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“…The regulatory ncRNA can control the expression of coding regions, and therefore, its proper functioning is vital for the viability of the organism. , Housekeeping noncoding RNAs like transfer RNA (tRNA) and ribosomal RNA (rRNA) are important components of protein translation machinery. Various coding and noncoding RNAs are depicted in Figure . - …”

Section: Introductionmentioning

confidence: 99%

“…Various coding and noncoding RNAs are depicted in Figure 1. [2][3][4][5][6] Initially identified as a messenger between DNA and proteins, RNA was considered a carrier of information from the nucleus to the cytoplasm, 7,8 apart from being the genetic material of certain viruses. Long treated as irrelevant to therapeutic intervention, RNA was termed "undruggable" owing to its structural degeneracy.…”

Section: Introductionmentioning

confidence: 99%

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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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