Quantitative evaluation of siRNA delivery in vivo

RNA. 2010 Dec;16(12):2553-63. doi: 10.1261/rna.2255810. Epub 2010 Oct 12.

Abstract

Effective small interfering RNA (siRNA)-mediated therapeutics require the siRNA to be delivered into the cellular RNA-induced silencing complex (RISC). Quantitative information of this essential delivery step is currently inferred from the efficacy of gene silencing and siRNA uptake in the tissue. Here we report an approach to directly quantify siRNA in the RISC in rodents and monkey. This is achieved by specific immunoprecipitation of the RISC from tissue lysates and quantification of small RNAs in the immunoprecipitates by stem-loop PCR. The method, expected to be independent of delivery vehicle and target, is label-free, and the throughput is acceptable for preclinical animal studies. We characterized a lipid-formulated siRNA by integrating these approaches and obtained a quantitative perspective on siRNA tissue accumulation, RISC loading, and gene silencing. The described methodologies have utility for the study of silencing mechanism, the development of siRNA therapeutics, and clinical trial design.

MeSH terms

  • Animals
  • Animals, Genetically Modified
  • Antibodies / isolation & purification
  • Antibodies / metabolism
  • Antibodies / pharmacology
  • Antibody Specificity
  • Argonaute Proteins
  • Cells, Cultured
  • Eukaryotic Initiation Factor-2 / immunology
  • Eukaryotic Initiation Factor-2 / metabolism
  • Evaluation Studies as Topic
  • Female
  • Gene Silencing / physiology
  • Gene Targeting / methods
  • Gene Transfer Techniques* / standards
  • Humans
  • Immunoprecipitation / methods
  • Immunoprecipitation / standards
  • Macaca mulatta
  • Mice
  • Mice, Inbred ICR
  • Protein Binding
  • RNA, Small Interfering / administration & dosage
  • RNA, Small Interfering / genetics*
  • RNA, Small Interfering / metabolism
  • Rats
  • Rats, Sprague-Dawley
  • Rodentia

Substances

  • AGO2 protein, human
  • Antibodies
  • Argonaute Proteins
  • Eukaryotic Initiation Factor-2
  • RNA, Small Interfering