Axonal transport of adeno-associated viral vectors is serotype-dependent

Gene Ther. 2013 Mar;20(3):348-52. doi: 10.1038/gt.2012.27. Epub 2012 Mar 15.

Abstract

We have previously shown that adeno-associated virus type 2 (AAV2) undergoes anterograde axonal transport in rat and non-human primate brain. We screened other AAV serotypes for axonal transport and found that AAV6 is transported almost exclusively in a retrograde direction and, in the same way as AAV2, it is also neuron-specific in rat brain. Our findings show that axonal transport of AAV is serotype dependent and this has implications for gene therapy of neurological diseases such as Huntington's disease.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Axonal Transport*
  • Brain / cytology
  • Brain / metabolism*
  • Corpus Striatum / cytology
  • Corpus Striatum / metabolism
  • Dependovirus / classification
  • Dependovirus / genetics*
  • Fluorescent Antibody Technique
  • Genetic Therapy / methods
  • Genetic Vectors / genetics
  • Green Fluorescent Proteins / genetics
  • Green Fluorescent Proteins / metabolism
  • Humans
  • Huntington Disease / genetics
  • Huntington Disease / therapy
  • Neurodegenerative Diseases / genetics
  • Neurodegenerative Diseases / therapy
  • Rats
  • Rats, Sprague-Dawley
  • Serotyping
  • Species Specificity
  • Thalamus / cytology
  • Thalamus / metabolism
  • Transduction, Genetic / methods*

Substances

  • Green Fluorescent Proteins