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.
Table of Contents
Section 1. Cellular Therapies1. Introduction to stem cell biology and its role in treating neurologic disorders
2. History of cellular grafting for CNS repair A clinical perspective
3. Autologous vs. heterologous cell replacement strategies for Parkinson disease and other neurologic diseases
Section 2. Gene therapies
4. Viral vectors and gene delivery to the central nervous system
5. Viral versus Non-viral approaches
Section 3. General Considerations (for cell and gene therapies)
6. Clinical/surgical considerations
7. Ethics of gene and cell therapy development for neurological disorders
8. Clinical trial designs and endpoints
9. Immunology of such therapies
10. Scaling of cell and gene therapies to population
11. The Health economics of cell and gene therapies
Section 4. Disease specific interventions using cells and genes
12. Disease specific interventions: The use of cell and gene therapies for Parkinson disease
13. Using gene or cell therapies to treat Huntington’s disease
14. Disorders of the motorneuron including ALS and SMA
15. Gene and cell-based therapies for retinal and optic nerve disease
16. Disease specific interventions using cell therapies for spinal cord disease/injury
17. Stem and progenitor cell-based therapy of myelin disorders
18. Cell and gene therapy in neuro-oncology
