目錄
總論 1
基因組編輯應(yīng)用和政策問題概述 2
人類基因組編輯的應(yīng)用 4
監(jiān)管人類基因組編輯的原則 10
建議 12
1 概述 14
研究現(xiàn)況 16
研究背景 19
研究方法 25
報(bào)告概要 25
2 人類基因組編輯監(jiān)管的總體原則 27
人類基因組編輯的監(jiān)管原則 27
美國(guó)基因治療技術(shù)的監(jiān)管 32
其他國(guó)家的監(jiān)管細(xì)節(jié) 53
總結(jié)和建議 53
3 基因組編輯技術(shù)的基礎(chǔ)研究應(yīng)用 57
基因組編輯基本方法 57
基因組編輯技術(shù)的飛速發(fā)展 63
人類細(xì)胞和組織基礎(chǔ)實(shí)驗(yàn)研究進(jìn)展 65
哺乳動(dòng)物繁殖與發(fā)育基礎(chǔ)實(shí)驗(yàn)研究進(jìn)展 66
基礎(chǔ)研究中的倫理和監(jiān)管問題 75
總結(jié)和建議 76
4 體細(xì)胞基因組編輯 78
研究背景 79
基因編輯較傳統(tǒng)基因治療和早期方案的優(yōu)勢(shì) 81
基于核酸酶的同源與非同源重組編輯修復(fù)技術(shù) 85
人類體細(xì)胞基因編輯技術(shù)的潛在應(yīng)用 86
基因編輯的設(shè)計(jì)和應(yīng)用涉及的科技考量 88
體細(xì)胞基因編輯產(chǎn)生的倫理和監(jiān)管問題 97
總結(jié)和建議 103
5 可遺傳性基因組編輯 105
潛在應(yīng)用方向和替代方法 107
相關(guān)的科學(xué)和技術(shù)問題 110
以糾正引發(fā)疾病性狀為目的的種系編輯帶來(lái)的倫理監(jiān)管問題 112
規(guī)則制定 123
總結(jié)和建議 126
6 基因強(qiáng)化 130
人類遺傳多樣性及“正�！焙汀白匀弧钡亩�義 131
了解公眾對(duì)基因強(qiáng)化的態(tài)度 132
如何區(qū)分基因治療和基因強(qiáng)化 137
種系( 遺傳) 基因組的編輯和強(qiáng)化 147
總結(jié)和建議 154
7 公眾參與 155
公眾參與：廣義概念 157
美國(guó)的實(shí)踐 160
國(guó)際上的實(shí)踐 164
公眾參與性研討活動(dòng)的經(jīng)驗(yàn) 166
更進(jìn)一步 167
總結(jié)和建議 168
8 原則和建議概要 171
人類基因組編輯監(jiān)管原則 171
美國(guó)監(jiān)督人類基因組編輯的現(xiàn)有機(jī)制 173
參考文獻(xiàn) 183
附錄A 基因組編輯的基礎(chǔ)科學(xué) 199
附錄B 國(guó)際研究監(jiān)管法規(guī) 236
附錄C 資料來(lái)源及方法 246
附錄D 組委會(huì)成員簡(jiǎn)介 251
附錄E 術(shù)語(yǔ)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