Section 1- Cellular Therapies1. Introduction to stem cell biologyo
Yamanaka, Gurdon, Rao, Thomson, Gage, Studer, Parmar
2. History of cellular graftingo
Lindvall/Bjorklund or Steve Dunnett
3. Allogenic vs Autologous approacheso
Federoff/Jun and Masayo Takahashi
Section 2- Gene therapies
4. Introduction to methods of gene transfero
Samulski, Xiao, Mulligan, Glorioso, Breakefield
5. History of gene therapy (and recombinant DNA)o
Verma, During, Wilson
6. Role the RAC (USA)o
Patterson, Corrigan-Curay
7. Viral vs Non-viral approacheso
Mart Saarma (Finland)
Section 3- General Considerations (and perhaps combine these sections for cell and gene therapies)
8. Clinical/surgical considerationso
Barker, Palfi, Tom Foltynie
9. Ethical considerationso
Charo, Macklin, Kaplan, Jonathan Sugarman
10. Manufacturing considerationso
Melissa Carpenter/Jacqueline Barry (Cell & Gene Therapy Catapult UK)
11. Clinical trial designs and clinical endpointso
Karl Kieburtz/ Tony Lang
12. Immunology of such therapieso
Hakan Widner; Emanuele Cozzi
13. Role of neuroimaging/biomarkerso
Paola Piccini/Jon Stoessel/David Eidelberg
14. Scaling to populationo
Andreas Bosio/Stefan Irions
15. Health economics and outcomeso
TBD
Section 4- Disease specific interventions using cells and genes
16. Parkinson’s Diseaseo
Barker
17. Huntington’s Disease o
Rosser
18. ALS o
Svendsen
19. Stroke o
Kakaia
20. AMD o
Coffey
21. Spinal cord o
Armin Curt
22. Retinitis Pigmentosa o
Ali
23. Demyelination o
Goldman
24. Epilepsy o
Kriegstein
Section 5- Gene Therapies.
25. Clinical/surgical considerationso
Larson, Bankiewicz, Kaplitt, Gross, Rothstein
26. Ethical considerationso
Kahn
27. Manufacturing considerationso
Robin Ali
28. Clinical trial designs and endpointso
Kieburtz, Olanow, Ravina
29. Role of neuroimaging/biomarkerso
Eidelberg, Stossel
30. Scaling to populationso
Lonser
31. Health economics and outcomeso
Roan-Gresenz, others