人类基因组编辑:科学·伦理·管理:science, ethics, and governance:中英对照

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