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DNA, RNA, and nucleoproteins constitute the fundamental molecular components responsible for the storage, transmission, and expression of genetic information in all living organisms [1]. DNA serves as the long-term storage of genetic blueprints, while RNA molecules, including mRNA and rRNA, facilitate the translation of these blueprints into functional proteins [1]. Nucleoproteins are complex assemblies, such as ribosomes and histones, that provide structural support and catalyze essential biological processes like protein synthesis and chromatin packaging [2]. In pharmacology, these molecules are major therapeutic targets; for example, alkylating agents and intercalators target DNA to treat various cancers by inducing apoptosis, while many antibiotics target bacterial ribosomes (nucleoproteins) to inhibit protein synthesis [3, 4]. Recent advancements have introduced RNA-targeted therapies, such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), which allow for the precise modulation of gene expression [5]. However, because these targets are ubiquitous and essential for normal cell function, drugs that interact with them often present significant safety concerns, including genotoxicity, myelosuppression, and the potential for secondary malignancies [6]. Sources: [1] Alberts B, et al. Molecular Biology of the Cell. [2] NIH-NHGRI. [3] PubChem-Cisplatin. [4] StatPearls-Ribosome-targeting antibiotics. [5] Nature Reviews Drug Discovery (2022). [6] FDA Guidance on Genotoxicity.
Drugs targeting these molecules act through various mechanisms including DNA alkylation, intercalation, inhibition of topoisomerases, ribosomal protein synthesis inhibition, and RNA interference or antisense modulation [3, 4, 5].
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