Cell transplantation and gene therapy in neurodegenerative disease /

Detalles Bibliográficos
Clasificación:Libro Electrónico
Otros Autores: Lane, Emma L., Drew, Cheney J. G., Lelos, Mariah J.
Formato: Electrónico eBook
Idioma:Inglés
Publicado: [S.l.] : Academic Press, 2022.
Colección:International review of neurobiology ; v. 166.
Temas:
Nervous system > Degeneration > Gene therapy.
Cell transplantation.
Acceso en línea:Texto completo
Tabla de Contenidos:
  • 4.1.3. Methods for generating a cell product for HD: Why is there variability in the output of PSC-derived MSN directed d ...
  • 4.1.4. Alternative neural cell types as CRT products: Does the cell product for transplantation need to be MSN precursors?
  • 4.2. Effective study of recovery
  • 4.2.1. Capturing successful behavioral recovery in animal models: Do we have the necessary tools to determine whether a g ...
  • 4.2.2. Mechanism of recovery: How long do we expect it to take for graft induced recovery to occur? How does graft-derive ...
  • 4.2.3. The impact of graft development and health: How do graft characteristics correlate with the capacity for functiona ...
  • 4.3. Wider challenges
  • 5. Conclusions and authors perspectives
  • References
  • Chapter Two: Challenges of cell therapies for retinal diseases
  • 1. Introduction
  • 2. Retinal disorders: Focus on AMD and RP
  • 2.1. Age-related macular degeneration
  • 2.2. Retinitis pigmentosa
  • 3. Human pluripotent stem cells for retinal cell therapy
  • 3.1. RPE cell therapy
  • 3.2. PR cell therapy
  • 4. Transposing bench-scale practices to industrialization
  • 4.1. Graf rejection: Immunological considerations
  • 4.2. Automation, cryopreservation and final formulation
  • 4.3. Cell function prediction using artificial intelligence
  • 5. Conclusion
  • Funding Statement
  • References
  • Chapter Three: Cell transplantation to repair the injured spinal cord
  • 1. Introduction
  • 2. Transplantation for spinal cord repair
  • 3. Pro-regenerative transplants: Bridging for spinal cord repair
  • 3.1. Peripheral nerve bridge
  • 3.2. Schwann cells
  • 3.3. Olfactory ensheathing cells
  • 3.4. Central glia: The astrocytes, oligodendrocytes, and their precursors
  • 3.5. Other pro-regenerative ``bridging�� transplants
  • 4. Building novel neuronal pathways: Neuronal relays for repair.
  • 4.1. Neural tissue transplantation
  • 4.2. Neural progenitor cells (NPCs)
  • 4.3. FSC-derived NPCs prepared without cell culture
  • 4.4. FSC-derived NPCs prepared using cell culture
  • 4.5. Neural stem and progenitor cells from other tissues
  • 4.6. Brain-derived NPCs
  • 4.7. NPCs derived from pluripotent stem cells
  • 4.8. iPSC-derived NPCs
  • 5. Cell transplantation for neuroimmune modulation and neuroprotection
  • 6. Considerations for contraindications and adverse effects
  • 7. Closing remarks
  • References
  • Further reading
  • Chapter Four: Investigating cell therapies in animal models of Parkinson�s and Huntington�s disease: Current challenges a ...
  • 1. Introduction
  • 2. Challenge 1: Identifying a good model for cell transplantation studies
  • 2.1. The healthy vs diseased brain
  • 2.2. Choosing the appropriate rodent species
  • 2.3. Choosing the appropriate strain
  • 2.4. Parkinson�s disease models for cell transplantation studies
  • 2.5. Huntington�s disease models for cell transplantation studies
  • 3. Challenge 2: Survival of xenografts in rodent models
  • 3.1. The use of immunosuppressants in immunocompetent hosts
  • 3.2. The use of immunodeficient rodents and rodents with humanized immune systems
  • 3.3. Neonatal desensitization to support graft survival
  • 4. Challenge 3: Assessing the functional efficacy of the graft
  • 4.1. General considerations for assessing the efficacy of a cell therapy
  • 4.2. Assessing the efficacy of a cell therapy product on cognitive function in models of PD and HD
  • 5. Challenge 4: Creating clinically relevant models
  • 5.1. Using young animals to model the aged brain
  • 5.2. Acknowledging complex transplant-drug interactions
  • 5.3. Identifying when brain size matters
  • 6. Discussion
  • References.
  • Chapter Five: Considerations for the use of biomaterials to support cell therapy in neurodegenerative disease
  • 1. Using biomaterials to support cell therapies
  • 2. Considerations for the use of biomaterials
  • 2.1. Material selection
  • 2.2. Material formulation and delivery
  • 2.3. Biocompatibility and degradation
  • 2.4. Mechanical properties and the host response
  • 3. Promising biomaterial and cell based therapies for neurodegenerative disease
  • 4. Clinical translation
  • 5. Conclusion
  • References
  • Chapter Six: Challenges of translating a cell therapy to GMP
  • 1. The history of cell and gene therapy manufacturing
  • 1.1. The bone marrow transplant experience
  • 1.2. The stem cell (gene) therapy experience
  • 1.3. Adverse events in gene therapy
  • 1.4. Advances in safer ex vivo gene therapy product manufacturing
  • 1.5. Good manufacturing practice
  • 1.6. Requirements for GMP manufacturing of cell and gene therapy products in the US
  • 1.7. GMP grade cell product development and manufacturing for neurological disorders
  • 2. Characterization of the NSCs
  • 2.1. Steps to consider for later phase GMP manufacturing of an NSC line
  • 2.2. Future direction of cell and gene therapy for neurological disorders
  • 2.3. A state of the art GMP facility for cell and gene therapy product manufacturing
  • 2.4. Example of a GMP facility for cell and gene therapy manufacturing at UC Davis
  • 2.5. UC Davis GMP facility air pressurization
  • 3. History and track record
  • 4. QC/QA at the UC Davis GMP facility
  • 4.1. Recruiting and training of personnel
  • 4.2. Conclusion
  • References
  • Chapter Seven: Considerations for clinical trial design and conduct in the evaluation of novel advanced therapeutics in n ...
  • 1. Introduction
  • 1.1. The potential use of cell and gene therapies in HD
  • 1.2. The potential use of cell and gene therapies for PD.
  • 2. Principles of clinical trial design
  • 2.1. Methods for reducing bias in clinical trials
  • 2.1.1. Randomization
  • 2.1.2. Allocation concealment
  • 2.1.3. Blinding
  • 2.2. Specific considerations for clinical trial design in the context of cell and gene therapies in neurodegeneration
  • 2.2.1. Addressing the problem of small numbers using within patient trial designs
  • 2.2.2. Minimizing bias in ATMP trials in neurodegenerative disease
  • 2.2.3. Alternative trial designs that negate the requirement for sham controls
  • 2.2.3.1. Use of historical control data
  • 2.2.3.2. The randomized start design
  • 2.2.3.3. Trials within a cohort
  • 3. Outcome measures in cell and gene therapy trials in neurodegenerative disease
  • 3.1. Outcomes applicable to cell and gene therapies in neurodegenerative disease
  • 3.1.1. Outcomes measures for use in cell and gene therapy trials with reference to HD and PD
  • 3.2. The development and utilization of core outcome sets
  • 3.3. Considerations for the use of biomarkers and surrogate endpoints
  • 4. Technical and practical factors for consideration in operationalizing the intracranial delivery of cell and gene therapies
  • 4.1. Devices for delivering cell and gene therapies to the brain
  • 4.2. Considerations for increasing the scale of cell and gene therapy trials in neurodegeneration
  • 4.2.1. Defining the surgical procedure
  • 4.2.2. Defining peri-operative requirements for trial delivery and patient care
  • 5. Considerations for the inclusion of participants in trials of cell and gene therapies
  • 5.1. Participant selection
  • 5.2. Communicating with participants in cell and gene therapy investigations
  • 5.3. Promoting diversity and inclusion across trial participants
  • 6. Summary
  • References.

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