Safety, Ethics and Regulations

Abstract

This invaluable resource discusses the saftey, ethics, and regulations of developing stem cell clinical applications. Each chapter is contributed by a preeminent scientist in the field and covers such topics as clinical safety of stem cell gene therapy, the patentability of hESC technologies, international guidelines, challenges to international stem cell clinical trials, worldwide regulations including in emerging markets like China and Taiwan. Saftey, Ethics, and Regulations and the other books in the Stem Cells in Clinical Applications series will be invaluable to scientists, researchers, advanced students and clinicians working in stem cells, regenerative medicine or tissue engineering. Preface; About the Editors; Contents; Contributors; Part I: Safety; Chapter 1: The Safety of Allogeneic Stem Cell Transplantation; 1.1 Introduction; 1.2 Stem Cell Sources; 1.2.1 Bone Marrow; 1.2.2 Peripheral Blood Stem Cells; 1.2.3 Umbilical Cord Blood; 1.3 Donor Selection and Testing; 1.3.1 HLA Restrictions; 1.3.2 Related Donor; 1.3.3 Unrelated Donor; 1.3.4 Cord Blood; 1.4 Reported Microbiological Transmissions; 1.4.1 Transmission of Human Immunodeficiency Virus Type 1 (HIV-1); 1.4.1.1 Prevention of HIV Transmission by a Viremic Donor; 1.4.2 Transmission of Hepatitis B Virus (HBV) 1.4.2.1 How to Proceed with Anti-HBc-Positive Donors?1.4.2.2 Prevention of HBV Infection in Case of HBV Viremic Donors; 1.4.3 Transmission of Hepatitis C Virus (HCV); 1.4.3.1 Prevention of HCV Transmission; 1.4.4 Transmission of HTLV-I + II; 1.4.5 Cytomegalic Virus (CMV) in CMV-Negative Recipients; 1.4.6 Possible Risks from Donors with Epstein-Barr Virus (EBV); 1.4.7 Transmission of Treponema pallidum; 1.4.8 Commensal Bacteria; 1.4.9 Other Pathogens; 1.5 Transfusion-Related Complications; 1.5.1 Transfusion-Related Acute Lung Injury (TRALI); 1.5.2 Hemolytic Transfusion Reaction (HTR) 1.6 Risk of Transmission of Donor-Derived Noninfectious Diseases1.6.1 Possible Long-Time Effects; 1.6.2 Donor-Derived Malignancies; 1.7 Conclusion; References; Chapter 2: Induced Pluripotent Stem Cell Therapy and Safety Concerns in Age-Related Chronic Neurodegenerative Diseases; 2.1 Overview of Age-Related Chronic Neurodegenerative Diseases; 2.1.1 Alzheimer's Disease; 2.1.2 Parkinson's Disease; 2.2 Induced Pluripotent Stem Cells Technology; 2.2.1 Reprogramming Methods; 2.2.1.1 Viral Vector-Based Methods; 2.2.1.2 Naked DNA-Based Methods; 2.2.1.3 Non-DNA-Based Methods; 2.3 Applications of iPSCs 2.3.1 Disease Modeling2.3.2 Drug Screening and Testing; 2.3.3 Cell Replacement Therapy; 2.4 Future Directions: Challenges and Advancements; 2.4.1 Limitations in iPSC Generation and Potential Solutions; 2.4.1.1 Integration into Genome; 2.4.1.2 Epigenetic Memory; 2.4.1.3 Differentiation and Purity; 2.4.1.4 Control Groups for Research; 2.4.2 Safety Concerns for Clinical Grade iPSCs; 2.4.2.1 Tumorigenicity, Immunogenicity, and Genomic Instability; 2.4.2.2 Biobanking; 2.4.2.3 Clearance of Animal Products; 2.4.3 Recent Biological and Biotechnological Advancements 2.4.3.1 Alternative Strategies for Reprogramming2.4.3.2 Three-Dimensional (3D) and Organoid Cultures; 2.4.3.3 Biotechnological Strategies; Biomaterials; Bioprinting; 2.5 Conclusion; References; Chapter 3: Clinical Safety and Applications of Stem Cell Gene Therapy; 3.1 Introduction; 3.2 Applications; 3.3 Safety; 3.3.1 Proof of Concept; 3.3.2 The Preclinical Testing Program; 3.3.3 Suicide Genes; 3.4 Efficacy; 3.4.1 Promoter Selection; 3.4.2 Transduction Efficiency; 3.4.3 Engraftment Efficiency; 3.5 Gene Therapy Procedures; 3.6 Way Forward; 3.7 Conclusion; References