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I22 Intranasal administration of mesenchymal stem cells ameliorates the abnormal dopamine transmission system and inflammatory reaction in the R6/2 mouse model of huntington disease
  1. Libo Yu-Taeger1,2,
  2. Janice Stricker-Shaver1,2,
  3. Katrin Arnold3,4,
  4. Patrycja Bambynek-Dziuk1,2,
  5. Arianna Novati1,2,
  6. Elisabeth Singer1,2,
  7. Fabian Claire3,4,
  8. Janine Magg1,2,
  9. Olaf Riess1,2,
  10. Alexandra Stolzing3,5,
  11. Lusine Danielyan6,
  12. Hoa Huu Phuc Nguyen1,2,7
  1. 1Institute of Medical Genetics and Applied Genomics, University of Tuebingen, Tuebingen, Germany
  2. 2Centre for Rare Diseases (ZSE), University of Tuebingen, Tuebingen, Germany
  3. 3Interdisciplinary Centre for Bioinformatics (IZBI), University of Leipzig, Germany
  4. 4Fraunhofer Institute for Cell Therapy and Immunology (IZI), Leipzig, Germany
  5. 5Centre for Biological Engineering, Wolfson School, Loughborough University, Loughborough, UK
  6. 6Department of Clinical Pharmacology, University of Tuebingen, Germany
  7. 7Department of Human Genetics, Ruhr University of Bochum Germany


Background Huntington disease (HD) is a fatal neurodegenerative disease that currently has no effective treatment. Although intrastriatal administration of mesenchymal stem cells (MSCs) has shown beneficial effects in HD rodent models, its invasive nature and potential host immune reactivity have undermined its clinical use. Hence, we aim to evaluate the non-invasive intranasal administration (INA) as an effective alternative route for delivering MSCs in HD.

Methods MSCs at passage 3 were intranasally administered to 4-week-old R6/2 mice and their survival rate, motor function and locomotor activities were evaluated until 11 weeks of age. Expression levels of inflammatory regulators and proteins involved in dopamine neurotransmission were analyzed using real-time PCR and Western blot in the 11-week-old mice.

Results When compared to PBS-treated R6/2 littermates, MSCs-treated R6/2 mice showed an increased survival rate, decreased body weight loss at a later disease stage and attenuated sleep disturbance. MSCs treatment also increased protein expression levels of DARPP-32 and tyrosine hydroxylase (TH) and restored increased gene expression of inflammatory modulators.

Conclusions Our results demonstrate significantly ameliorated phenotypes of R6/2 mice after MSCs administration via INA. This indicates that INA is an effective delivering route of MSCs to the brain for HD therapy.

  • dopamine
  • Huntington disease
  • inflammation
  • intranasal
  • mesenchymal stem cells
  • R6/2 mice

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