Lentiviral-mediated gene transfer of siRNAs for the treatment of Huntington's disease

Methods Mol Biol. 2013:1010:95-109. doi: 10.1007/978-1-62703-411-1_7.

Abstract

This chapter describes the potential use of viral-mediated gene transfer in the central nervous system for the silencing of gene expression using RNA interference in the context of Huntington's disease (HD). Protocols provided here describe the design of small interfering RNAs, their encoding in lentiviral vectors (LVs) and viral production, as well as procedures for their stereotaxic injection in the rodent brain.

MeSH terms

  • Animals
  • Brain / metabolism
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Humans
  • Huntingtin Protein
  • Huntington Disease / genetics*
  • Huntington Disease / therapy
  • Lentivirus / genetics*
  • Mice
  • Mutation
  • Nerve Tissue Proteins / deficiency
  • Nerve Tissue Proteins / genetics
  • RNA, Small Interfering / genetics*

Substances

  • HTT protein, human
  • Huntingtin Protein
  • Nerve Tissue Proteins
  • RNA, Small Interfering