The trigeminal retrograde transfer pathway in the treatment of neurodegeneration

Stephanos Kyrkanides, Meixiang Yang, Ross H. Tallents, Jen nie H. Miller, Sabine M. Brouxhon, John A. Olschowka

Research output: Contribution to journalArticlepeer-review

10 Scopus citations


The trigeminal sensory system was evaluated for the retrograde transfer of gene therapy vectors into the CNS. The feline immunodeficiency viral vector, FIV(HEXB), encoding for the human HEXB gene, was injected intra-articularly in the temporomandibular joint of 12 week-old HexB-/- mice displaying clinical and histopathological signs of Sandhoff disease. This treatment regiment reduced GM2 storage and ameliorated neuroinflammation in the brain of HexB-/- mice, as well as attenuated behavioral deficits. In conclusion, retrograde transfer along trigeminal sensory nerves may prove to be a valuable route of gene therapy administration for the treatment of lysosomal storage disorders and other neurodegenerative diseases.

Original languageEnglish
Pages (from-to)139-142
Number of pages4
JournalJournal of Neuroimmunology
Issue number1-2
StatePublished - Apr 30 2009

Bibliographical note

Funding Information:
This work was funded in part by grant NS048339 from the National Institutes of Health.


  • GM gangliosidosis
  • Mouse, Gene therapy
  • Sandhoff disease
  • β-hexosaminidase

ASJC Scopus subject areas

  • Immunology and Allergy
  • Immunology
  • Neurology
  • Clinical Neurology


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