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Updated: May 15, 2026

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
10:33

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

siRNA delivery: from basics to therapeutic applications

Tiziana Musacchio1, Vladimir P Torchilin

  • 1Center for Pharmaceutical Biotechnology and Nanomedicine, Northeastern University, 360 Huntington Ave, Boston, MA 02115, USA.

Frontiers in Bioscience (Landmark Edition)
|January 2, 2013
PubMed

Insights

Small interfering RNA (siRNA) offers therapeutic potential for gene-dependent diseases but faces delivery challenges. This review explores advanced strategies to enhance siRNA stability, delivery, and efficacy for clinical applications.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • Small interfering RNA (siRNA) holds promise for treating gene-dependent diseases.
  • Current limitations include poor pharmacokinetics, short half-life, low in vivo stability, rapid renal clearance, and inefficient transfection.

Purpose of the Study:

  • To review advanced strategies for overcoming siRNA delivery and efficacy challenges.
  • To discuss the therapeutic prospects of improved siRNA formulations.

Main Methods:

  • Literature review of recent advancements in siRNA delivery systems.
  • Analysis of novel approaches to enhance stability and transfection efficiency.

Main Results:

  • Various strategies are being developed to improve siRNA pharmacokinetics and stability.
  • Enhanced delivery systems show potential for increased gene silencing efficacy.

Conclusions:

  • Addressing siRNA's limitations is crucial for its successful therapeutic application.
  • Continued innovation in delivery technologies will unlock siRNA's full clinical potential.

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