Cell and Gene Therapies for Neurologic Diseases
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Cell and Gene Therapies for Neurologic Diseases
Federoff, Howard J.; Barker, Roger A.
Elsevier - Health Sciences Division
10/2024
340
Dura
Inglês
9780323901208
15 a 20 dias
Descrição não disponível.
Section 1. Cellular Therapies
1. 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
1. 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
Este título pertence ao(s) assunto(s) indicados(s). Para ver outros títulos clique no assunto desejado.
AAV; Adrenal medulla; Allogeneic; Alpha-synuclein; Amyotrophic lateral sclerosis; Antigen presentation; ATMP; Autologous; Bioethics; Blood-brain barrier; Brain disorders; Brain tissue transplantation; CDNF; Cell- and tissue-based therapy; Cell manufacturing; Cell therapies; Cell therapy; Cell transplant; Cell transplantation; Centralized manufacturing; Clinical considerations; Clinical trial; Clinical trials; Cost utility analysis; Decentralized manufacturing; Discounting; Disease modeling; Disease modification; Dopamine; Dopamine neurons; Dyskinesias; Embryonic stem cells; Endpoint; ER stress; Feasibility; Fetal tissue; First-in-human trials; GDNF; Gene regulation; Gene therapies; Gene therapy; Genetic modifiers; Genetic therapy; Glaucoma; Glial progenitor cell; Glioblastoma; Grafts; Healthcare access; Heterologous; High-grade glioma; Highly specialized technology evaluation; HSV-1; Human organoid; Huntingtin-lowering; Huntington disease; Huntington's disease; Immune privilege; Immunity; Immunization; Induced pluripotent stem cells; Industrialization; Informed consent; In-process control; iPSC; Leukodystrophy; MANF; Multiple sclerosis; Myelin; Nanoparticles; Nerve regeneration; Neurodegeneration; Neurturin; Oligonucleotide; Onasemnogene abeparvovec; Optic nerve diseases; Outcome assessment; Outscaling; Parkinson; Parkinson disease; Parkinson's disease; PDGF; Pharmacoeconomics; Pluripotent stem cells; Positron emission tomography; Process automation; Process integration; Quality control; Regeneration; Remyelination; Retinal degeneration; Retinal diseases; Risk; Safety; Spinal cord injury; Stem cell; Stem cell therapy; Stem cells; Striatum; Surgical considerations; Tissue-specific stem cells; Transplantation; Trial design; UPR; Upscaling; Viral vectors
Section 1. Cellular Therapies
1. 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
1. 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
Este título pertence ao(s) assunto(s) indicados(s). Para ver outros títulos clique no assunto desejado.
AAV; Adrenal medulla; Allogeneic; Alpha-synuclein; Amyotrophic lateral sclerosis; Antigen presentation; ATMP; Autologous; Bioethics; Blood-brain barrier; Brain disorders; Brain tissue transplantation; CDNF; Cell- and tissue-based therapy; Cell manufacturing; Cell therapies; Cell therapy; Cell transplant; Cell transplantation; Centralized manufacturing; Clinical considerations; Clinical trial; Clinical trials; Cost utility analysis; Decentralized manufacturing; Discounting; Disease modeling; Disease modification; Dopamine; Dopamine neurons; Dyskinesias; Embryonic stem cells; Endpoint; ER stress; Feasibility; Fetal tissue; First-in-human trials; GDNF; Gene regulation; Gene therapies; Gene therapy; Genetic modifiers; Genetic therapy; Glaucoma; Glial progenitor cell; Glioblastoma; Grafts; Healthcare access; Heterologous; High-grade glioma; Highly specialized technology evaluation; HSV-1; Human organoid; Huntingtin-lowering; Huntington disease; Huntington's disease; Immune privilege; Immunity; Immunization; Induced pluripotent stem cells; Industrialization; Informed consent; In-process control; iPSC; Leukodystrophy; MANF; Multiple sclerosis; Myelin; Nanoparticles; Nerve regeneration; Neurodegeneration; Neurturin; Oligonucleotide; Onasemnogene abeparvovec; Optic nerve diseases; Outcome assessment; Outscaling; Parkinson; Parkinson disease; Parkinson's disease; PDGF; Pharmacoeconomics; Pluripotent stem cells; Positron emission tomography; Process automation; Process integration; Quality control; Regeneration; Remyelination; Retinal degeneration; Retinal diseases; Risk; Safety; Spinal cord injury; Stem cell; Stem cell therapy; Stem cells; Striatum; Surgical considerations; Tissue-specific stem cells; Transplantation; Trial design; UPR; Upscaling; Viral vectors
