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The emergence of gene and cellular therapeutics has changed the clinical landscape for a variety of disorders and is now ready to do so for neurological diseases. These therapeutic modalities currently complement and may in time supplant small molecule drugs. This volume comprehensively covers the scientific background, translational efforts, clinical developments and registered biologics that have entered into clinical practice. Coverage includes types of therapies available and in development, and best practice uses for a variety of neurological disorders including Parkinson s, Huntington s, ALS, stroke, spinal cord RP, demyelination, and epilepsy.
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 Dunnett3. Allogenic vs Autologous approacheso Federoff/Jun and Masayo TakahashiSection 2- Gene therapies4. Introduction to methods of gene transfero Samulski, Xiao, Mulligan, Glorioso, Breakefield5. History of gene therapy (and recombinant DNA)o Verma, During, Wilson6. Role the RAC (USA)o Patterson, Corrigan-Curay7. 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 Foltynie9. Ethical considerationso Charo, Macklin, Kaplan, Jonathan Sugarman10. Manufacturing considerationso Melissa Carpenter/Jacqueline Barry (Cell & Gene Therapy Catapult UK)11. Clinical trial designs and clinical endpointso Karl Kieburtz/ Tony Lang12. Immunology of such therapieso Hakan Widner; Emanuele Cozzi13. Role of neuroimaging/biomarkerso Paola Piccini/Jon Stoessel/David Eidelberg14. Scaling to populationo Andreas Bosio/Stefan Irions15. Health economics and outcomeso TBDSection 4- Disease specific interventions using cells and genes16. Parkinson s Diseaseo Barker17. Huntington s Disease o Rosser18. ALS o Svendsen19. Stroke o Kakaia20. AMD o Coffey21. Spinal cord o Armin Curt22. Retinitis Pigmentosa o Ali23. Demyelination o Goldman24. Epilepsy o Kriegstein Section 5- Gene Therapies.25. Clinical/surgical considerationso Larson, Bankiewicz, Kaplitt, Gross, Rothstein26. Ethical considerationso Kahn27. Manufacturing considerationso Robin Ali28. Clinical trial designs and endpointso Kieburtz, Olanow, Ravina29. Role of neuroimaging/biomarkerso Eidelberg, Stossel30. Scaling to populationso Lonser31. Health economics and outcomeso Roan-Gresenz, others