reviewMolecular TherapyJan 9, 2024HYBRID OA

The landscape of nanoparticle-based siRNA delivery and therapeutic development

University of Greenwich · Beijing Institute of Technology · +2 more institutions

PubMed
Indexed incrossrefpubmed

Abstract

Five small interfering RNA (siRNA)-based therapeutics have been approved by the Food and Drug Administration (FDA), namely patisiran, givosiran, lumasiran, inclisiran, and vutrisiran. Besides, siRNA delivery to the target site without toxicity is a big challenge for researchers, and naked-siRNA delivery possesses several challenges, including membrane impermeability, enzymatic degradation, mononuclear phagocyte system (MPS) entrapment, fast renal excretion, endosomal escape, and off-target effects. The siRNA therapeutics can silence any disease-specific gene, but their intracellular and extracellular barriers limit their clinical applications. For this purpose, several modifications have been employed to siRNA…

Citation impact

177
total citations
FWCI
37.13
Percentile
100%
References
300
Citations per year

Authors

6

Topics & keywords

Keywords
  • Small interfering RNA
  • Nanocarriers
  • Transfection
  • RNA interference
  • Chemistry
  • Gene delivery
  • Liposome
  • Drug delivery
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