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|a Federoff, Howard J.,
|e author.
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| 245 |
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|a Cell and Gene Therapies for Neurologic Diseases /
|c Howard J. Federoff and Roger A. Barker.
|
| 250 |
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|a First edition.
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| 264 |
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1 |
|a Amsterdam, Netherlands :
|b Elsevier,
|c [2024]
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|c ©2024
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|a 1 online resource (631 pages).
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|a Handbook of Clinical Neurology Series ;
|v Volume 205
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| 588 |
|
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|a Description based on publisher supplied metadata and other sources.
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| 505 |
0 |
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|a Intro -- Cell and Gene Therapies for Neurologic Diseases -- Copyright -- Handbook of Clinical Neurology 3rd Series -- Foreword -- Preface -- Contributors -- Contents -- Section I: Cellular therapies -- Chapter 1: Introduction to stem cell biology and its role in treating neurologic disorders -- Introduction to Stem Cells -- Stem cells in life and regeneration -- Pluripotent stem cells -- Cell Replacement Using Pluripotent Stem Cells -- Single cell-type cell replacement in the brain -- Stem cell-based circuit reconstruction in the brain -- Transplant Immunology Applied to Stem Cell Neural Grafts in the Brain -- Allogeneic or autologous grafting-Cell intrinsic factors -- Allogeneic or autologous grafting-Cell extrinsic factors -- New possibilities for mediating graft rejection in the stem cell era -- Universal, immune evasive cells -- Reprogramming in Regenerative Neuroscience -- Direct conversion into induced neurons -- In vivo conversion -- References -- Chapter 2: History of cellular grafting for central nervous system repair-A clinical perspective -- Introduction -- Preclinical Work in Parkinson Model Triggered the First Clinical Interest -- First Clinical Trials With Cellular Grafting: The Rise and Fall of Adrenal Medulla Transplantation in PD -- Translation of Human Fetal Dopaminergic Neurons Toward the Clinic -- Open-Label Clinical Trials With Human Fetal Dopaminergic Neurons: First Evidence of Surviving and Functional Grafts in P -- Major Setback for Cellular Grafting by Sham Surgery-Controlled Clinical Trials With Human Fetal Dopamine Neurons -- Other Cell Sources For Dopaminergic Neuron Replacement: No Clinical Success -- New Insight Into The Pathogenesis of PD by Human Fetal Dopamine Grafts -- Revival of the Field of Cellular Grafting and Clinical Translation of Stem Cells -- What Can We Learn From the History of Cellular Grafting?.
|
| 505 |
8 |
|
|a Why Have GDNF and NRTN Trials Had Modest Clinical Outcomes? -- Future Directions -- Acknowledgments -- References -- Section III: General considerations (for cell and gene therapies) -- Chapter 6: Clinical/surgical considerations -- Clinical Factors -- Precision of diagnosis -- Disease heterogeneity -- Stage of disease -- Consent and capacity -- Comorbidity -- Other inclusion and exclusion criteria -- Pregnancy and conception -- Concomitant medications -- Acceptability of trial design -- Surgical Factors -- Advanced product delivery options -- Practices to date -- Specific instruments -- FHC STARDRIVE -- Duration of administration-Acceptability -- Simultaneous bilateral administration -- Documenting effects and adverse effects -- Neurosurgical Factors Relevant to Trial Design -- References -- Chapter 7: Ethics of gene and cell therapy development for neurologic disorders -- Introduction -- Conclusion and Future Prospects -- References -- Chapter 8: Clinical trial designs and endpoints -- An Introduction into Trial Design -- Patient and Public Engagement in Trial Design -- Trial Inclusivity and Diversity -- Strategies for engaging cultural and ethnic minorities -- Using national health service data as a recruitment tool -- Determining Sample Size -- Defining Study Populations -- Participant identification challenges -- Randomization, Stratification and Minimization -- Control Arms -- The placebo effect -- Ethical considerations -- Types of controls -- Clinical Endpoints for Gene and Cell Therapy Trials -- Efficacy endpoints -- Measures of target engagement and establishing optimal dosing -- Safety endpoints -- Concluding Remarks -- References -- Chapter 9: Immunology of cell and gene therapy approaches for neurologic diseases -- Introduction -- Basic Premise of How the Immune System and the Brain Interact -- Critical Factors of the Afferent Arm.
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| 505 |
8 |
|
|a Tissue incompatibility -- Pre-exposure status of the recipient of a cellular graft or gene therapy -- Repeated treatments -- Mitigation steps -- Cell Therapy Procedures -- Gene therapy -- Mitigation steps -- Tissue composition, antigen-presenting cells -- Vascularization, origin of endothelium -- Mitigation steps -- Mode of implantation into the brain of cells and vectors -- Mitigation steps -- Immunization -- Mitigation steps -- Factors and Properties of the Efferent Arm of the Immune Response -- Homing of activated lymphocytes to the site of inflammation -- Localized homing -- Mitigation steps -- Blood-brain barrier properties -- Mitigation steps -- Immunosuppression -- Duration of treatment -- Immunosuppressive Regimens in the Ongoing Clinical Trials With Stem Cell-Derived Cell Products for Parkinson's Dis -- Detection of Immune Responses or Graft Rejection -- Rejection treatment -- Summary -- References -- Chapter 10: Scaling of cell and gene therapies to population -- Introduction -- Treatment Schemes/Regimes and Their Implications for Manufacturing Concepts and Scales -- Pragmatism of Early Clinical Trials in Regenerative Medicine -- From Clinical Development to Commercial Phase -- Industrializing Cell Manufacturing -- References -- Chapter 11: The health economics of cell and gene therapies -- Introduction -- Value-Based Assessment -- Cost utility analysis -- Economic perspective -- Discounting -- Highly specialized technology evaluation -- Evidence Generation Challenges -- Management of uncertainty -- Outcomes-based reimbursement -- Affordability -- Future Directions -- References -- Section IV: Disease specific interventions using cells and genes -- Chapter 12: Disease-specific interventions: The use of cell and gene therapies for Parkinson disease -- Introduction -- Cell Therapy Approaches For Treating PD.
|
| 500 |
|
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|a Chapter 14: Cell and gene therapy for amyotrophic lateral sclerosis.
|
| 520 |
|
|
|a This volume, part of the 'Handbook of Clinical Neurology' series, focuses on the application of cell and gene therapies in the treatment of neurological diseases. Edited by Howard J. Federoff and Roger A. Barker, it compiles the latest research and methodologies in the field, providing insights into innovative treatments for conditions affecting the nervous system. The book is designed for clinicians, researchers, and students in neurology, offering detailed discussions on therapies, their development, and clinical applications. It aims to advance understanding and improve therapeutic practices in neurology through cutting-edge scientific contributions.
|
| 588 |
|
|
|a Part of the metadata in this record was created by AI, based on the text of the resource.
|
| 588 |
|
|
|a Description based on print version record.
|
| 504 |
|
|
|a Includes bibliographical references and index.
|
| 650 |
|
0 |
|a Nervous system
|x Diseases.
|
| 650 |
|
2 |
|a Nervous System Diseases
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| 650 |
|
6 |
|a Système nerveux
|x Maladies.
|
| 655 |
|
7 |
|a Electronic books.
|2 local
|
| 700 |
1 |
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|a Barker, Roger A.,
|e author.
|
| 710 |
2 |
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|a ScienceDirect (Online service)
|
| 830 |
|
0 |
|a Handbook of clinical neurology ;
|v Volume 205.
|
| 856 |
4 |
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|u http://proxy.library.tamu.edu/login?url=https://www.sciencedirect.com/science/handbooks/00729752/205
|z Connect to the full text of this electronic book
|t 0
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| 880 |
8 |
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|6 505-00
|a References -- Chapter 3: Autologous vs heterologous cell replacement strategies for Parkinson disease and other neurologic diseases -- Introduction -- Clinical Substrate for CNS Transplantation: Parkinson Disease -- Cell Replacement Strategies Deployed in PD Clinical Trials -- Derivation of PSCs -- Differentiation of the Midbrain Dopaminergic Lineage and Proof-of-Principle Studies -- Autologous Vs Allogeneic Approach -- Preclinical Autologous Vs Heterologous Grafts in Nonhuman Primates -- Strategies for Immunosuppression and Immune Cloaking of Cells -- Final Thoughts -- References -- Section II: Gene therapies -- Chapter 4: Viral vectors for gene delivery to the central nervous system -- Introduction -- AAV Vectors -- Vector design and transgene expression -- Vector delivery and cell transduction -- AAV-related immunity and toxicity -- Adenoviral (AdV) vectors -- Background and vector design strategies -- Toxicity and immunogenicity of AdV vectors in humans -- AdV for the CNS -- γ-Retroviral (γ-RV) and Lentiviral Vectors (LV) -- Background -- Vector design and CNS transduction -- Improvements in vector engineering to address safety, integration, and transgene expression -- HSV-1 Vectors -- HSV biology and replication-defective vector design -- Replication-defective vector production -- Modifications to enhance the duration and specificity of transgene expression in the CNS -- HSV amplicon (HSV-amp) vector design and production -- Amplicon vectors for CNS applications -- Clinical Trial Approaches for CNS Disorders -- Conclusions -- References -- Chapter 5: Viral and nonviral approaches -- Introduction and Problems in Parkinson Disease Treatment -- Characteristics of different ways of biopharmaceutical administration into the brain -- Virus Vectors -- Protein Delivery -- GDNF and NRTN -- Platelet-derived growth factor -- CDNF and MANF.
|
| 880 |
8 |
|
|6 505-00
|a Human fetal ventral mesencephalic tissue (hfVM) transplants -- Non-hfVM, nonstem cell tissue grafts used in trials for PD -- Stem cell-derived dopamine cells -- The next generation of stem cell-derived dopamine cells -- Gene Therapy (GT) Approaches for Treating PD -- Vectors-Coming of age -- Adeno-associated viruses (AAVs) -- Lentiviruses (LVs) -- Strategies for gene therapy in PD -- DA-synthetic GTs-Correcting the striatal dopamine deficit -- Lentiviral (EIAV) triple enzyme -- AAV-mediated AADC approach -- GTs that modulate circuit activity -- GTs that modify disease progression: Neurotrophic factors -- Neurturin -- GDNF -- CDNF -- GTs that modify disease progression: Other molecular strategies -- Reducing levels of α-synuclein protein -- Vectorized antibodies against α-synuclein -- α-Synuclein gene repression -- Overexpression of wild-type genes to enhance lysosomal or mitochondrial function -- GBA1 -- PARKIN -- Potential hurdles for all GT strategies -- Targeting -- Timing -- Conclusions -- References -- Chapter 13: Using gene or cell therapies to treat Huntington´s disease -- Introduction -- How Gene Therapies Could Be Used to Treat HD -- Huntingtin-lowering approaches -- Genetic modifier approaches -- Manipulating downstream cell processes -- Prospects and Challenges for Gene Therapies in HD -- DNA-targeting strategies -- RNA-targeting strategies -- Allele selectivity -- Biomarkers in gene therapy trials -- Delivery -- How Cell Therapies Could Be Used to Treat HD -- Transplantation of MSN progenitor cells -- Proof-of-concept -- State-of-the-art -- Transplantation of glial cells -- Proof-of-concept and state-of-the-art -- Transplantation of cells for neural support -- Proof-of-concept and state-of-the-art -- Neural stem cells -- Mesenchymal stem cells -- Engineered cells -- Prospects and Challenges for Cell Therapies in HD -- References.
|
| 955 |
|
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|a Elsevier ScienceDirect 2026-2027
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| 994 |
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