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Trans-neuronal transduction of spinal neurons following cortical injection and anterograde axonal transport of a bicistronic AAV1 vector

Research output: Contribution to journalArticlepeer-review

T. H. Hutson, C. Kathe, L. D F Moon

Original languageEnglish
Pages (from-to)231-236
Number of pages6
JournalGene Therapy
Issue number2
Early online date10 Dec 2015
Accepted/In press3 Nov 2015
E-pub ahead of print10 Dec 2015
Published1 Feb 2016

King's Authors


Adeno-associated viral (AAV) vectors are one of the most promising gene delivery systems to the central nervous system. We now report, that AAV1 can be used to express transgenes trans-neuronally in neurons distant from the injection site. Specifically, intracortical injection of a bicistronic AAV1 vector trans-neuronally transduced spinal neurons as shown by fluorescence microscopy, the presence of AAV genome and AAV transcript in the contralateral spinal cord. Prior pyramidotomy abolished spinal transduction, confirming anterograde axonal transport of AAV1 in the corticospinal tract. These observations demonstrate the potential of bicistronic AAV1 for trans-neuronal expression of therapeutic transgenes in neurological disorders or reporter genes in connectivity studies.

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