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RNA interference (RNAi) has revolutionized the field of gene regulation by enabling targeted posttranscriptional silencing through small RNA molecules such as siRNAs, miRNAs, shRNAs, and piRNAs. Since its discovery in Caenorhabditis elegans, RNAi has become a versatile tool in functional genomics, disease modeling, drug design, and therapeutic development. RNAi-based therapeutics are evolving rapidly, with four siRNA drugs already receiving U.S. Food and Drug Administration (FDA) approval and numerous others currently under clinical trial. This chapter provides a comprehensive overview of RNAi mechanisms, including the roles of endogenous miRNAs, exogenous siRNAs, and the RNA-induced silencing complex (RISC). It also discusses delivery challenges, addressing physiological and pharmacokinetic barriers, and compares viral and nonviral delivery systems. Additionally, this chapter also reviews RNAi-based therapeutic advancements, ongoing clinical trials, and its role in precision medicine and personalized therapy. Emerging technologies such as CRISPR-RNAi systems, AI-guided RNAi design, and self-assembled RNA constructs are also explored for their potential to enhance specificity and therapeutic efficacy.

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RNAi in the Postgenomic Era: Delivery, Drug Design, and Emerging Technologies

  • 章节

  • 发表日期：

    2026年

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  • Shyamalima Saikia,
  • Indrani Gogoi,
  • Damini Dey,
  • Karpumpuli Mili,
  • Ankita Purkayastha,
  • Kaushik Kumar Mech,
  • Rajdeep Bora,
  • Kistina Neog,
  • Pankaj Chetia,
  • Minakshi Puzari

摘要

RNA interference (RNAi) has revolutionized the field of gene regulation by enabling targeted posttranscriptional silencing through small RNA molecules such as siRNAs, miRNAs, shRNAs, and piRNAs. Since its discovery in Caenorhabditis elegans, RNAi has become a versatile tool in functional genomics, disease modeling, drug design, and therapeutic development. RNAi-based therapeutics are evolving rapidly, with four siRNA drugs already receiving U.S. Food and Drug Administration (FDA) approval and numerous others currently under clinical trial. This chapter provides a comprehensive overview of RNAi mechanisms, including the roles of endogenous miRNAs, exogenous siRNAs, and the RNA-induced silencing complex (RISC). It also discusses delivery challenges, addressing physiological and pharmacokinetic barriers, and compares viral and nonviral delivery systems. Additionally, this chapter also reviews RNAi-based therapeutic advancements, ongoing clinical trials, and its role in precision medicine and personalized therapy. Emerging technologies such as CRISPR-RNAi systems, AI-guided RNAi design, and self-assembled RNA constructs are also explored for their potential to enhance specificity and therapeutic efficacy.