Advances in lipid-based platforms for RNAi therapeutics

Sara Falsini, Laura Ciani, Sandra Ristori, Angelo Fortunato, Annarosa Arcangeli

Research output: Contribution to journalReview articlepeer-review

18 Scopus citations

Abstract

Sequence-specific gene silencing, known as RNA interference (RNAi), is a natural process that can be exploited for knocking-down specific genes involved in the insurgence/development of pathological processes. In 2001 the discovery that small interfering RNA (siRNA) can induce gene silencing without immunoresponse turned RNAi into a promising technique for the control of post-transcriptional gene expression. Nowadays, the major challenge remains infusion in vivo. Therefore, vehicles providing protection and selective transport are to be developed for efficient systemic delivery. The most used vectors are lipid-based, offering a wide range of biocompatible formulations. Here their application in molecular medicine is discussed, especially with regard to recent clinical trials where conventional therapies have failed. The role played by extended physicochemical characterization for the success of RNAi therapeutics is also evidenced.

Original languageEnglish (US)
Pages (from-to)1138-1146
Number of pages9
JournalJournal of Medicinal Chemistry
Volume57
Issue number4
DOIs
StatePublished - Feb 27 2014
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Medicine
  • Drug Discovery

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