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Related Concept Videos

siRNA - Small Interfering RNAs02:30

siRNA - Small Interfering RNAs

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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Site-Targeted Drug Delivery Systems: Polymeric Carriers01:24

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Polymeric carriers enhance targeted drug delivery by increasing efficacy while minimizing off-target effects. These carriers comprise a biodegradable polymeric backbone integrated with functional elements that enable targeting, improve physicochemical properties, and regulate drug release.Targeting MechanismsThe targeting ability of polymeric carriers is mediated by a homing device, which is a molecular recognition component designed to selectively bind to specific tissues or cells. Monoclonal...
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Experimental RNAi02:15

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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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Preparation of Neutrally-charged, pH-responsive Polymeric Nanoparticles for Cytosolic siRNA Delivery
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Lipid nanoparticle delivery systems for siRNA-based therapeutics.

C Wan1, T M Allen, P R Cullis

  • 1Department of Biochemistry and Molecular Biology, University of British Columbia, Vancouver, Canada, cw7h@mail.ubc.ca.

Drug Delivery and Translational Research
|March 20, 2015
PubMed
Summary
This summary is machine-generated.

New ionizable lipid nanoparticles (LNPs) effectively deliver small interfering RNA (siRNA) therapeutics in vivo. These advanced LNP siRNA systems demonstrate potent gene silencing and promising clinical results for various diseases.

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Area of Science:

  • Biochemistry
  • Molecular Biology
  • Nanotechnology

Background:

  • Small interfering RNA (siRNA) therapeutics offer significant disease treatment potential but require effective in vivo delivery systems.
  • Lipid nanoparticles (LNPs) are established drug delivery vehicles, with prior success in delivering small molecules.

Purpose of the Study:

  • To develop advanced LNP systems for efficient and safe in vivo delivery of siRNA therapeutics.
  • To overcome challenges associated with cationic lipids, such as toxicity and rapid clearance.

Main Methods:

  • Design and synthesis of ionizable cationic lipids with specific pKa values for pH-dependent charge characteristics.
  • Optimization of LNPs to enhance endosomal escape and maximize gene silencing efficacy.
  • Evaluation of LNP siRNA systems in preclinical mouse models and clinical trials.

Main Results:

  • Developed ionizable cationic lipids enabling efficient oligonucleotide encapsulation at low pH and neutral surface charge at physiological pH.
  • Achieved potent gene silencing in hepatocytes at low doses (0.005 mg siRNA/kg) in mouse models.
  • Demonstrated clinical efficacy for treating hypercholesterolemia and transthyretin-induced amyloidosis.

Conclusions:

  • Ionizable LNP siRNA systems represent a significant advancement in nucleic acid therapeutics delivery.
  • These optimized LNP siRNA formulations show high efficacy and safety, paving the way for broader clinical applications.
  • Future development is expected to yield more LNP siRNA therapeutics targeting diverse diseases and tissues.