目录 总论 1 基因组编辑应用和政策问题概述 2 人类基因组编辑的应用 4 监管人类基因组编辑的原则 10 建议 12 1 概述 14 研究现况 16 研究背景 19 研究方法 25 报告概要 25 2 人类基因组编辑监管的总体原则 27 人类基因组编辑的监管原则 27 美国基因治疗技术的监管 32 其他国家的监管细节 53 总结和建议 53 3 基因组编辑技术的基础研究应用 57 基因组编辑基本方法 57 基因组编辑技术的飞速发展 63 人类细胞和组织基础实验研究进展 65 哺乳动物繁殖与发育基础实验研究进展 66 基础研究中的伦理和监管问题 75 总结和建议 76 4 体细胞基因组编辑 78 研究背景 79 基因编辑较传统基因治疗和早期方案的优势 81 基于核酸酶的同源与非同源重组编辑修复技术 85 人类体细胞基因编辑技术的潜在应用 86 基因编辑的设计和应用涉及的科技考量 88 体细胞基因编辑产生的伦理和监管问题 97 总结和建议 103 5 可遗传性基因组编辑 105 潜在应用方向和替代方法 107 相关的科学和技术问题 110 以纠正引发疾病性状为目的的种系编辑带来的伦理监管问题 112 规则制定 123 总结和建议 126 6 基因强化 130 人类遗传多样性及“正常”和“自然”的定义 131 了解公众对基因强化的态度 132 如何区分基因治疗和基因强化 137 种系( 遗传) 基因组的编辑和强化 147 总结和建议 154 7 公众参与 155 公众参与：广义概念 157 美国的实践 160 国际上的实践 164 公众参与性研讨活动的经验 166 更进一步 167 总结和建议 168 8 原则和建议概要 171 人类基因组编辑监管原则 171 美国监督人类基因组编辑的现有机制 173 参考文献 183 附录A 基因组编辑的基础科学 199 附录B 国际研究监管法规 236 附录C 资料来源及方法 246 附录D 组委会成员简介 251 附录E 术语表 264 Contents Summary 1 OVERVIEW OF GENOME-EDITING APPLICATIONS AND POLICY ISSUES 2 APPLICATIONS OF HUMAN GENOME EDITING 4 PRINCIPLES TO GUIDE THE GOVERNANCE OF HUMAN GENOME EDITING 10 RECOMMENDATIONS 12 1 Introduction 14 STUDY CONTEXT 16 BACKGROUND 19 STUDY APPROACH 25 ORGANIZATION OF THE REPORT 26 2 Oversight of Human Genome Editing and Overarching Principles for Governance 27 PRINCIPLES FOR GOVERNANCE OF HUMAN GENOME EDITING 27 REGULATION OF GENE THERAPY IN THE UNITED STATES 32 GOVERNANCE IN OTHER NATIONS 55 CONCLUSIONS AND RECOMMENDATION 56 3 Basic Research Using Genome Editing 57 THE BASIC TOOLS OF GENOME EDITING 57 RAPID ADVANCES IN GENOME-EDITING TECHNOLOGY 63 BASIC LABORATORY RESEARCH TO ADVANCE UNDERSTANDING OF HUMAN CELLS AND TISSUES 65 BASIC LABORATORY RESARCH TO ADVANCE UNDERSTANDING OF MAMMALIAN REPRODUCTION AND DEVELOPMENT 66 ETHICAL AND REGULATORY ISSUES IN BASIC RESEARCH 76 CONCLUSIONS AND RECOMMENDATION 77 4 Somatic Genome Editing 78 BACKGROUND 79 ADVANTAGES OF GENOME EDITING OVER TRADITIONAL GENE THERAPY AND EARLIER APPROACHES 81 HOMOLOGOUS AND NONHOMOLOGOUS REPAIR METHODS USED FOR NUCLEASE-BASED GENOME EDITING 85 POTENTIAL HUMAN APPLICATIONS OF SOMATIC CELL GENOME EDITING 86 SCIENTIFIC AND TECHNICAL CONSIDERATIONS ASSOCIATED WITH THE DESIGN AND APPLICATION OF GENOME-EDITING STRATEGIES 88 ETHICAL AND REGULATORY ISSUES POSED BY SOMATIC CELL GENOME EDITING 97 CONCLUSIONS AND RECOMMENDATIONS 103 5 Heritable Genome Editing 105 POTENTIAL APPLICATIONS AND ALTERNATIVES 107 SCIENTIFIC AND TECHNICAL ISSUES 110 ETHICS AND REGULATION OF EDITING THE GERMLINE TO CORRECT DISEASE-CAUSING TRAITS 112 REGULATION 123 CONCLUSIONS AND RECOMMENDATION 127 6 Enhancement 130 HUMAN GENETIC VARIATION AND DEFINING “NORMAL” AND “NATURAL” 131 UNDERSTANDING PUBLIC ATTITUDES TOWARD ENHANCEMENT 132 DRAWING LINES: THERAPY VERSUS ENHANCEMENT 137 GERMLINE (HERITABLE) GENOME EDITING AND ENHANCEMENT 147 CONCLUSIONS AND RECOMMENDATIONS 154 7 Public Engagement 155 PUBLIC ENGAGEMENT: BROAD CONCEPTS 157 U.S. PRACTICES 160 INTERNATIONAL PRACTICES 164 LESSONS LEARNED FROM PUBLIC ENGAGEMENT ACTIVITIES 166 MOVING FORWARD 168 CONCLUSIONS AND RECOMMENDATIONS 169 8 Summary of Principles and Recommendations 171 OVERARCHING PRINCIPLES FOR GOVERNANCE OF HUMAN GENOME EDITING 171 EXISTING U.S. OVERSIGHT MECHANISMS FOR HUMAN GENOME EDITING 173 References 183 Appendix A The Basic Science of Genome Editing 199 Appendix B International Research Oversight and Regulations 236 Appendix C Data Sources and Methods 246 Appendix D Committee Member Biographies 251 Appendix E Glossary 264
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