MicroRNA-regulated, systemically delivered rAAV9: a step closer to CNS-restricted transgene expression

Mol Ther. 2011 Mar;19(3):526-35. doi: 10.1038/mt.2010.279. Epub 2010 Dec 21.

Abstract

Recombinant adeno-associated viruses (rAAVs) that can cross the blood-brain-barrier and achieve efficient and stable transvascular gene transfer to the central nervous system (CNS) hold significant promise for treating CNS disorders. However, following intravascular delivery, these vectors also target liver, heart, skeletal muscle, and other tissues, which may cause untoward effects. To circumvent this, we used tissue-specific, endogenous microRNAs (miRNAs) to repress rAAV expression outside the CNS, by engineering perfectly complementary miRNA-binding sites into the rAAV9 genome. This approach allowed simultaneous multi-tissue regulation and CNS-directed stable transgene expression without detectably perturbing the endogenous miRNA pathway. Regulation of rAAV expression by miRNA was primarily via site-specific cleavage of the transgene mRNA, generating specific 5' and 3' mRNA fragments. Our findings promise to facilitate the development of miRNA-regulated rAAV for CNS-targeted gene delivery and other applications.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Cell Line, Tumor
  • Central Nervous System / metabolism*
  • Dependovirus / genetics*
  • Gene Expression Regulation / genetics
  • Gene Silencing
  • Gene Transfer Techniques
  • Genes, Reporter / genetics
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics*
  • HEK293 Cells
  • Humans
  • Male
  • Mice
  • Mice, Inbred C57BL
  • MicroRNAs / genetics
  • MicroRNAs / metabolism*
  • Models, Biological
  • Molecular Sequence Data
  • Sequence Alignment
  • Transgenes* / genetics

Substances

  • MicroRNAs