Cell and gene therapy for Parkinson’s disease

Deniz Kirik, MD, PhD
Professor of Neuroscience

Brain Repair And Imaging in...
Major symptoms of Parkinson’s disease




                             • Difficulty to initiate movement
                  ...
Parkinson’s disease is characterized by degeneration of
dopamine producing cells located in the substantia nigra
Parkinson’s disease is characterized by degeneration of
dopamine producing cells located in the substantia nigra
Kirik et al., 2005
Threshold dose of L-DOPA for inducing dyskinesia
decreases over duration of the disease


                                ...
Restorative treatment strategy for PD
  No of DA neurons




Onset of
symptoms

                                          ...
Striatal dopamine replacement strategy by fetal nigral
dopamine neurons

     Parkinson’s Disease                Fetal nig...
Gene therapy for DOPA delivery




                                 Carlsson et al., 2007
Fetal Cell Transplantation Procedure




                Ventral
                mesencephalon




                       ...
Fetal Cell Transplantation Procedure




                                       Thompson et al., 2005
Transplantation of human fetal DA neuroblasts




                                      Mendez et al., 2005
Grafts of fetal dopamine neurons survive and improve motor
                function in Parkinson's disease

              ...
History of Cell Transplantation in Parkinson's Disease



1979: First reports of functional restoration in
      rodent mo...
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Cell and gene therapy for Parkinson’s disease - part 1

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Presentation by Prof Deniz Kirik, MD, PhD at the Parkinson's UK Research Conference, November 2010 in York.

With introduction by Dr Oliver Bandmann.

Part 2: http://www.slideshare.net/ParkinsonsResearchUK/cell-and-gene-therapy-for-parkinsons-disease-part-2.

Part 3: http://www.slideshare.net/ParkinsonsResearchUK/cell-and-gene-therapy-for-parkinsons-disease-part-3

Published in: Health & Medicine
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Cell and gene therapy for Parkinson’s disease - part 1

  1. 1. Cell and gene therapy for Parkinson’s disease Deniz Kirik, MD, PhD Professor of Neuroscience Brain Repair And Imaging in Neural Systems (BRAINS) Unit Department of Experimental Medical Science Lund University, Sweden
  2. 2. Major symptoms of Parkinson’s disease • Difficulty to initiate movement • Slowness of movement • Stiffness • Shaking at rest • Postural imbalance
  3. 3. Parkinson’s disease is characterized by degeneration of dopamine producing cells located in the substantia nigra
  4. 4. Parkinson’s disease is characterized by degeneration of dopamine producing cells located in the substantia nigra
  5. 5. Kirik et al., 2005
  6. 6. Threshold dose of L-DOPA for inducing dyskinesia decreases over duration of the disease 1.8 1.2 L-dopa level (mg/kg) 0.8 0.4 0 Never Stable Wearing-o On-o treated responders (n=8) (n=6) (n=13) (n=12) Mouradian et al. 1987
  7. 7. Restorative treatment strategy for PD No of DA neurons Onset of symptoms Functional recovery Disease progression Restorative therapy
  8. 8. Striatal dopamine replacement strategy by fetal nigral dopamine neurons Parkinson’s Disease Fetal nigral transplantation Input Input Response Response Motor Motor output output
  9. 9. Gene therapy for DOPA delivery Carlsson et al., 2007
  10. 10. Fetal Cell Transplantation Procedure Ventral mesencephalon Transplantation Trypsin/DNase Mechanical dissociation
  11. 11. Fetal Cell Transplantation Procedure Thompson et al., 2005
  12. 12. Transplantation of human fetal DA neuroblasts Mendez et al., 2005
  13. 13. Grafts of fetal dopamine neurons survive and improve motor function in Parkinson's disease Lindvall et al., Science 1990
  14. 14. History of Cell Transplantation in Parkinson's Disease 1979: First reports of functional restoration in rodent models of PD 1990: First report of therapeutic effects in PD patients in open-label trials 2001: Reports of the first placebo-controlled double-blind studies
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