Small interfering RNA
Small interfering RNA (siRNA) is a short double-stranded RNA molecule that mediates sequence-specific gene silencing through the RNA interference (RNAi) pathway.
Small interfering RNA (siRNA) is a short double-stranded RNA molecule that mediates sequence-specific gene silencing through the RNA interference (RNAi) pathway. After cellular uptake, the siRNA duplex is loaded into the RNA-induced silencing complex (RISC). The antisense strand is retained, principally by the catalytic protein Argonaute 2 (AGO2), and guides the complex to a complementary messenger RNA (mRNA). Target recognition can promote cleavage and degradation of the mRNA, thereby reducing production of the encoded protein. Because siRNA acts at the level of gene expression, it can be used to investigate gene function and to target disease-associated proteins that may be difficult to inhibit with conventional small molecules or antibodies.
The principal pharmaceutical challenge is delivery. Naked siRNA is susceptible to degradation and has limited access to the cytoplasm, so therapeutic systems commonly use lipid nanoparticles, liposomes, polymeric nanoparticles, dendrimers, peptides, extracellular vesicles, or membrane-derived nanoplatforms. Current research applies siRNA-mediated gene knockdown to cancer, viral infection, pulmonary disease, and placental vascular disorders. Combinations with ferroptosis, apoptosis, photodynamic or photothermal therapy, and immune or epigenetic interventions are being investigated to increase therapeutic efficacy while controlling effects in healthy tissues.
Rebuilt from PubMed 18 Sept 2026 · no new papers today
Where the papers sit
13 papers study small interfering rna directly. Those 13 are one subject: Targeted RNA Therapeutics. Delivery engineering is expanding from peptide and polymeric nanoparticles to microneedles, exosomes, and metal-organic frameworks, enabling tissue-targeted gene silencing. Applications span psoriasis, HBV, glaucoma, and cancer, with ferroptosis and drug resistance recurring endpoints. No way of splitting those 13 scores better than chance.
Recent Findings on small interfering RNA
Targeted RNA Therapeutics: siRNA platforms increasingly pair sequence-specific gene silencing with localized or cell-selective delivery across inflammatory, viral, ocular, pulmonary, placental, and malignant disease models 42735507Sep42709589Sep42687852Sep42572107Aug42385857Jul. Engineered carriers—including dissolving microneedles, exosomes, macrophage membranes, lipid nanoparticles, polymeric nanoparticles, metal-organic frameworks, peptide nanocomplexes, and dendriplexes—improve tissue access, cytosolic delivery, retention, or target specificity 42687852Sep42679356Sep42572107Aug42425239Jul42398835Jul. Combination regimens amplify silencing through dexamethasone, photothermal ablation, sonodynamic therapy, ferroptosis, sorafenib, or photodynamic therapy, with sequential HBV treatment outperforming concurrent administration and monotherapy 42735507Sep42709589Sep42679356Sep42546831Aug42528415Jul42309466Jun. Molecular engineering is also refining asymmetric siRNA for AGO2 binding, divalent siRNA for simultaneous targets, membrane fusion for lysosomal bypass, and peptide systems that may expand delivery beyond liver tissues 42612768Aug42385857Jul42541443Aug. The results remain formulation-dependent: higher guanidinium/phosphate ratios increased airway-cell toxicity, while a glycodendrimer vector retained antitumor activity independently of siRNA; studies therefore emphasize selective, programmable, and safer carriers for broader clinical translation 42572107Aug42425239Jul42541443Aug.
Written from 13 PubMed abstracts, each one cited by PMID above. Published: 2026-09-11. Last written: 2026-09-16 by GPT. Drafted by language models from published abstracts; not medical advice.