Cell and Gene Therapies for Neurologic Diseases, Volume 205 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. As 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.
Section 1- Cellular Therapies1. Introduction to stem cell biologyo
2. History of cellular graftingo
3. Allogenic vs Autologous approacheso
Section 2- Gene therapies
4. Introduction to methods of gene transfero
5. History of gene therapy (and recombinant DNA)o
6. Role the RAC (USA)o
7. Viral vs Non-viral approacheso
Section 3- General Considerations (and perhaps combine these sections for cell and gene therapies)
8. Clinical/surgical considerationso
9. Ethical considerationso
10. Manufacturing considerationso
11. Clinical trial designs and clinical endpointso
12. Immunology of such therapieso
13. Role of neuroimaging/biomarkerso
14. Scaling to populationo
15. Health economics and outcomeso
Section 4- Disease specific interventions using cells and genes
16. Parkinson’s Diseaseo
17. Huntington’s Disease o
18. ALS o
19. Stroke o
20. AMD o
21. Spinal cord o
22. Retinitis Pigmentosa o
23. Demyelination o
24. Epilepsy o
Section 5- Gene Therapies.
25. Clinical/surgical considerationso
26. Ethical considerationso
27. Manufacturing considerationso
28. Clinical trial designs and endpointso
29. Role of neuroimaging/biomarkerso
30. Scaling to populationso
31. Health economics and outcomeso
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