Design of synthetic materials for intracellular delivery of RNAs: From siRNA-mediated gene silencing to CRISPR/Cas gene editing

Jason B. Miller, Daniel J. Siegwart

Research output: Contribution to journalReview articlepeer-review

29 Scopus citations

Abstract

Ribonucleic acids (RNAs) possess great therapeutic potential and can be used to treat a variety of diseases. The unique biophysical properties of RNAs, such as high molecular weight, negative charge, hydrophilicity, low stability, and potential immunogenicity, require chemical modification and development of carriers to enable intracellular delivery of RNAs for clinical use. A variety of nanomaterials have been developed for the effective in vivo delivery of short/ small RNAs, messenger RNAs, and RNAs required for gene editing technologies including clustered regularly interspaced palindromic repeat (CRISPR)/Cas. This review outlines the challenges of delivering RNA therapeutics, explores the chemical synthesis of RNA modifications and carriers, and describes the efforts to design nanomaterials that can be used for a variety of clinical indications.

Original languageEnglish (US)
Pages (from-to)5310-5337
Number of pages28
JournalNano Research
Volume11
Issue number10
DOIs
StatePublished - Oct 1 2018

Keywords

  • RNA interference (RNAi)
  • clustered regularly interspaced palindromic repeat (CRISPR)/Cas
  • messenger RNA (mRNA)
  • nanoparticles
  • nucleic acid therapeutics
  • synthetic nanomaterials

ASJC Scopus subject areas

  • General Materials Science
  • Electrical and Electronic Engineering

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