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|a Cell transplantation and gene therapy in neurodegenerative disease /
|c Edited by Emma L. Lane, Cheney J.G. Drew, Mariah J. Lelos.
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| 260 |
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|a [S.l.] :
|b Academic Press,
|c 2022.
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| 300 |
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|a 1 online resource.
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| 336 |
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|a text
|2 rdacontent
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|a online resource
|2 rdacarrier
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| 490 |
1 |
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|a International review of neurobiology ;
|v v. 166
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| 504 |
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|a Includes bibliographical references.
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8 |
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|a 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.
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| 505 |
8 |
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|a 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.
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| 505 |
8 |
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|a 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.
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| 505 |
8 |
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|a 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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| 650 |
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|a Nervous system
|x Degeneration
|x Gene therapy.
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| 650 |
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0 |
|a Cell transplantation.
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| 650 |
|
7 |
|a Cell transplantation.
|2 fast
|0 (OCoLC)fst00850240
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| 700 |
1 |
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|a Lane, Emma L.
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| 700 |
1 |
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|a Drew, Cheney J. G.
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| 700 |
1 |
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|a Lelos, Mariah J.
|
| 776 |
0 |
8 |
|i ebook version :
|z 9780323989381
|
| 776 |
0 |
8 |
|c Original
|z 0323989373
|z 9780323989374
|w (OCoLC)1310394910
|
| 776 |
0 |
8 |
|i Print version:
|t CELL TRANSPLANTATION AND GENE THERAPY IN NEURODEGENERATIVE DISEASE.
|d [S.l.] : ELSEVIER ACADEMIC PRESS, 2022
|z 0323989373
|w (OCoLC)1310394910
|
| 830 |
|
0 |
|a International review of neurobiology ;
|v v. 166.
|
| 856 |
4 |
0 |
|u https://sciencedirect.uam.elogim.com/science/bookseries/00747742/166
|z Texto completo
|
