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The Resource Gene Therapy and Cell Therapy Through the Liver : Current Aspects and Future Prospects

Gene Therapy and Cell Therapy Through the Liver : Current Aspects and Future Prospects

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Gene Therapy and Cell Therapy Through the Liver : Current Aspects and Future Prospects
Title
Gene Therapy and Cell Therapy Through the Liver
Title remainder
Current Aspects and Future Prospects
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Subject
Language
eng
Cataloging source
MiAaPQ
Literary form
non fiction
Nature of contents
dictionaries
Gene Therapy and Cell Therapy Through the Liver : Current Aspects and Future Prospects
Label
Gene Therapy and Cell Therapy Through the Liver : Current Aspects and Future Prospects
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http://libproxy.rpi.edu/login?url=https://ebookcentral.proquest.com/lib/rpi/detail.action?docID=4179001
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Copyright
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Carrier category
online resource
Carrier category code
cr
Carrier MARC source
rdacarrier
Color
multicolored
Content category
text
Content type code
txt
Content type MARC source
rdacontent
Contents
  • Foreword -- Preface -- Contents -- Chapter 1: Liver-Targeted Gene and Cell Therapies: An Overview -- 1.1 Gene Therapies -- 1.1.1 Liver-Directed Gene Delivery -- 1.1.2 Gene Therapy Strategies for Liver Disease Treatment -- 1.1.2.1 Gene Replacement -- 1.1.2.2 Gene Repair -- 1.1.2.3 Gene Augmentation -- 1.1.2.4 DNA Vaccination -- 1.1.2.5 Gene Silencing -- 1.2 Cell Therapies -- 1.2.1 Cell Sources -- 1.2.1.1 Primary Hepatocytes -- 1.2.1.2 Immortalized (Transformed) Hepatocytes -- 1.2.1.3 Stem Cells -- 1.2.1.4 Xenogeneic Cells -- 1.2.2 Cell Therapy Strategies for Liver Diseases Treatment -- 1.2.2.1 Liver Cell Transplantation -- 1.2.2.2 Extracorporeal Bioactive Liver Perfusion Systems -- 1.2.2.3 Liver Tissue Engineering -- 1.3 Future Perspectives -- References -- Part I: Cell Therapy -- Chapter 2: Macrophage Therapy for Liver Fibrosis and Regeneration -- 2.1 Introduction -- 2.2 Basic Research into the Biology of Macrophages During Liver Injury -- 2.2.1 The Dual Role of Macrophage During Liver Damage -- 2.2.2 Injection of Differentiated Bone Marrow-Derived Macrophages Can Improve Liver Fibrosis, Regeneration and Function in Mouse Models of Liver Fibrosis -- 2.2.3 TWEAK Produced by Macrophage Induces Expansion of the Ductular Reaction Containing Hepatic Progenitor Cells -- 2.2.4 Macrophages Are Key Players in Determining the Fate of Hepatic Progenitor Cells -- 2.3 Towards Macrophage Therapy -- References -- Chapter 3: Liver Regeneration Therapy Using Autologous Bone Marrow-Derived Cells for Cirrhotic Patients -- 3.1 Background -- 3.2 Trends in Liver Regeneration Therapy -- 3.3 Our Autologous Bone Marrow Cell Infusion Therapy (ABMi Therapy) Using Non-cultured Autologous Whole- Bone Marrow Cells (BMCs) -- 3.3.1 Inclusion Criteria -- 3.3.2 Exclusion Criteria -- 3.3.3 Protocol -- 3.3.4 Primary Endpoint -- 3.3.5 Secondary Endpoints
  • 3.4 Our Less-Invasive Liver Regeneration Therapy Using Cultured Autologous Bone Marrow-Derived Mesenchymal Stem Cells (BMSCs) -- 3.4.1 Inclusion Criteria -- 3.4.2 Protocol -- 3.4.3 Protocol Summary -- 3.4.4 Primary Endpoint -- 3.4.5 Secondary Endpoints -- 3.5 Conclusion -- References -- Chapter 4: Novel Immunotherapy Using Liver-Derived Natural Killer Cells for Preventing Hepatocellular Carcinoma Recurrence in Liver Transplantation -- 4.1 Introduction -- 4.2 Immunotherapy Using NK Cells -- 4.2.1 Characteristics of NK Cells -- 4.2.2 NK Immunotherapy for Cancers -- 4.3 Cytotoxic Potential of Liver-Derived NK Cells -- 4.3.1 Liver-Derived NK Cells in Mice -- 4.3.2 Liver NK Cells in Human -- 4.4 Clinical-Scale Isolation of Liver NK Cells from Donor Liver Graft -- 4.4.1 Protocol of NK Isolation -- 4.5 Clinical Trials for Living and Deceased Donor Liver Transplantation -- References -- Chapter 5: Cell-Based Immunotherapy for HCC: Our Experiences and Future Directions -- 5.1 Limitation of Conventional Treatments and Advantage of Immunotherapy for Intermediate to Advanced HCC -- 5.2 The Current Cell-Based Immunotherapy Trials -- 5.3 DC-Based Immunotherapy for HCC -- 5.4 Strategies to Enhance the Effects of DC-Based Cancer Vaccine -- 5.5 Future Perspective -- References -- Part II: Gene Therapy -- Chapter 6: AAV Vector-Mediated Liver Gene Therapy and Its Implementation for Hemophilia -- 6.1 Preface -- 6.2 General Features of AAV -- 6.3 A History of AAV Vector Development -- 6.3.1 Discovery and Vector Development of AAV1-6 (First Generation: Naturally Occurring Serotypes) -- 6.3.2 AAV Serotypes Discovered from Monkey and Human Tissues (Second Generation: Selected Naturally Occurring Serotypes and Clones) -- 6.3.3 Development of Novel Vectors Suitable for Specific Purposes (Third Generation: Ongoing) -- 6.4 A Brief History of Hemophilia Gene Therapy
  • 6.4.1 Overview of Hemophilia -- 6.4.2 Advantages of Gene Therapy as a Therapeutic Modality -- 6.4.3 Attempts Using Various Vectors for Hemophilia A -- 6.4.4 Attempts Using AAV2 Vectors for Hemophilia B -- 6.4.5 Clinical Trials Incorporating the AAV8 Vector -- 6.5 Considerations for Optimizing AAV Vector Administration -- 6.5.1 General Considerations -- 6.5.2 Liver-Mediated Gene Transfer -- 6.5.3 Skeletal Muscle Transduction -- 6.5.3.1 Direct Injection into Muscle -- 6.5.3.2 Other Methods for Muscle Transduction -- 6.5.3.3 Limb Perfusion -- 6.6 Neutralizing Antibody (NAb) Against AAV Vector -- 6.6.1 The Impact of NAb Upon AAV-Mediated Gene Transfer -- 6.6.2 Prevalence of NAb Against AAV Serotypes Around the World -- 6.6.3 Analysis of Japanese Population -- 6.6.4 Approaches for Evading Inhibitory Actions of NAb -- 6.6.5 Saline Flushing -- 6.7 The Search for the Best Vector for Liver Transduction -- 6.7.1 Early Results Using AAV2 -- 6.7.2 Features of AAV8 Vectors -- 6.7.3 Revival of AAV3-Based Vectors -- 6.8 Issues Regarding Empty Capsid -- 6.8.1 Characteristics of Empty Capsid -- 6.8.2 Impact of Empty Capsids Within Vector Preparations -- 6.8.3 Procedures of Removing Empty Capsid -- 6.9 Future Perspectives: Hurdles for Application -- 6.9.1 Difficulties in Translation -- 6.9.2 Preparation of Vectors for Human Trials -- 6.9.3 Standardization of Protocols -- 6.10 Concluding Remarks -- References -- Chapter 7: Potential Usage of Human Artificial Chromosome for Regenerative Medicine -- 7.1 Human Artificial Chromosome (HAC) as Episomal Vector -- 7.2 Microcell-Mediated Chromosome Transfer (MMCT) -- 7.3 HACs and Potential Medical Applications -- References -- Chapter 8: Image-Guided Hydrodynamic Gene Delivery to the Liver: Toward Clinical Applications -- 8.1 Introduction -- 8.2 Image-Guided Hydrodynamic Gene Delivery to the Liver
  • 8.2.1 Principles of Hydrodynamic Delivery -- 8.2.2 Image-Guided Hydrodynamic Delivery -- 8.2.3 Development of Clinically Applicable Injection Device -- 8.3 Conclusion -- References -- Part III: Legal Regulation -- Chapter 9: The Way to Clinical Application of Human Pluripotent Stem Cells -- 9.1 Pluripotent Stem Cells -- 9.2 Acceleration of iPS/ES Cell-Based Regenerative Medicine -- 9.3 Promising Projects for iPS/ES Cell-Based Regenerative Medicine -- 9.4 Concerns About the Clinical Application of Human Pluripotent Stem Cells -- 9.5 Past Regulatory Frameworks for Tissue/Cell-Based Therapy in Japan -- 9.6 Issues in the Past Regulatory Framework in Japan -- 9.7 Recent Regulatory Reform of Regenerative Medicine in Japan -- 9.8 Other Activities of Japanese Regulatory Authorities to Facilitate Regenerative Medicine Using Pluripotent Stem Cells -- 9.9 International Harmonization -- 9.10 Conclusion -- References -- Chapter 10: Regulation for Gene and Cell Therapy Medicinal Products in Europe -- 10.1 Introduction -- 10.2 Procedures for ATMPs Within the European Regulatory Pathway -- 10.2.1 Definitions and Classification -- 10.2.2 Certification -- 10.2.3 Scientific Advice at European and National Level -- 10.2.4 Marketing Authorization -- 10.2.5 Clinical Trials and "Hospital Exemption" -- 10.3 Regulatory Considerations Regarding Quality, Nonclinical and Clinical Testing -- 10.3.1 Procurement of Tissues and Cells and Traceability -- 10.3.2 Quality and Manufacturing Aspects of ATMPs -- 10.3.3 Nonclinical Studies for ATMPs -- 10.3.4 Aspects of Clinical Development of ATMPs -- 10.3.4.1 Cell-Based ATMPs -- 10.3.4.2 Gene Therapy Medicinal Products -- 10.3.5 Environmental Risks of Medicinal Products Containing or Consisting of Genetically Modified Organisms -- 10.4 Challenges and Future Directions -- References
  • Chapter 11: iPSC-Derived Products: Current Regulations -- 11.1 Introduction -- 11.2 Regulatory Issues with PSC-Based Therapy -- 11.3 The Japanese Regulations -- 11.4 US Efforts to Accelerate Therapy -- 11.5 European Regulations for Advanced Therapeutic Intervention -- 11.6 Summary -- References -- Chapter 12: Critical Path Initiative for Regenerative Medicine in Japan -- 12.1 Introduction -- 12.2 The Pharmaceuticals and Medical Devices Law (Revised Pharmaceutical Affairs Law) -- 12.3 The Act on the Safety of Regenerative Medicine -- 12.3.1 Circumstances of the Enactment -- 12.3.2 Outline of the Act -- 12.4 Conclusion -- References -- Part IV: Industrialization -- Chapter 13: Aseptic Manufacturing of Regenerative Medicine Products Using Isolator Technology -- 13.1 Introduction -- 13.2 The Art and Science of Aseptic Processing: A Brief Historical Perspective -- 13.2.1 Quality Control of Sterile Drugs -- 13.2.2 Risk and Sterility -- 13.2.3 Effectiveness of Gowns -- 13.3 Isolator Technology -- 13.3.1 What Is an Isolator? -- 13.3.2 General Features of an Isolator -- 13.3.3 The Isolator: A Very Brief History -- 13.4 Development of Room Decontamination Technology -- 13.4.1 Room Decontaminant and Disinfectant -- 13.4.2 Decontamination by Vapor Phase Hydrogen Peroxide (VPHP) -- 13.4.3 Development of Vapor Hydrogen Peroxide Decontamination System -- 13.4.4 Decontamination Efficacy -- 13.5 Application of the Isolator to Cell Culture Facilities for Regenerative Medicine -- 13.5.1 Development of Cell Culture-Related Technologies -- 13.5.2 Three-Dimensional Cell Incubation -- 13.5.3 Development of Cell Incubation Isolator, CPi, and CellPROi, an Automatic Cell Incubation System -- 13.6 Summary -- References -- Chapter 14: Cell and Vector Production Facility for Gene Therapy and Cell Therapy -- 14.1 Introduction
  • 14.2 Worldwide State of Biologics Manufacturing Facilities
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1 online resource (183 pages)
Form of item
online
Isbn
9784431556664
Media category
computer
Media MARC source
rdamedia
Media type code
c
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remote

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