Genome editing is a powerful new tool for making precise alterations to an organism's genetic material. Recent scientific advances have made genome editing more efficient, precise, and flexible than ever before. These advances have spurred an explosion of interest from around the globe in the possible ways in which genome editing can improve human health. The speed at which these technologies are being developed and applied has led many policymakers and stakeholders to express concern about whether appropriate systems are in place to govern these technologies and how and when the public should be engaged in these decisions.

Human Genome Editing considers important questions about the human application of genome editing including: balancing potential benefits with unintended risks, governing the use of genome editing, incorporating societal values into clinical applications and policy decisions, and respecting the inevitable differences across nations and cultures that will shape how and whether to use these new technologies. This report proposes criteria for heritable germline editing, provides conclusions on the crucial need for public education and engagement, and presents 7 general principles for the governance of human genome editing.

Contents

• The National Academies of SCIENCES • ENGINEERING • MEDICINE
• COMMITTEE ON HUMAN GENE EDITING: SCIENTIFIC, MEDICAL, AND ETHICAL CONSIDERATIONS
• Dedication
• Reviewers
• Preface
• Summary
  • OVERVIEW OF GENOME-EDITING APPLICATIONS AND POLICY ISSUES
  • APPLICATIONS OF HUMAN GENOME EDITING
  • PRINCIPLES TO GUIDE THE GOVERNANCE OF HUMAN GENOME EDITING
  • RECOMMENDATIONS
• 1. Introduction
  • STUDY CONTEXT
  • BACKGROUND
  • STUDY APPROACH
  • ORGANIZATION OF THE REPORT
• 2. Oversight of Human Genome Editing and Overarching Principles for Governance
  • PRINCIPLES FOR GOVERNANCE OF HUMAN GENOME EDITING
  • REGULATION OF GENE THERAPY IN THE UNITED STATES
  • GOVERNANCE IN OTHER NATIONS
  • CONCLUSIONS AND RECOMMENDATION
• 3. Basic Research Using Genome Editing
  • THE BASIC TOOLS OF GENOME EDITING
  • RAPID ADVANCES IN GENOME-EDITING TECHNOLOGY
  • BASIC LABORATORY RESEARCH TO ADVANCE UNDERSTANDING OF HUMAN CELLS AND TISSUES
  • BASIC LABORATORY RESEARCH TO ADVANCE UNDERSTANDING OF MAMMALIAN REPRODUCTION AND DEVELOPMENT
  • ETHICAL AND REGULATORY ISSUES IN BASIC RESEARCH
  • CONCLUSIONS AND RECOMMENDATION
• 4. Somatic Genome Editing
  • BACKGROUND
  • ADVANTAGES OF GENOME EDITING OVER TRADITIONAL GENE THERAPY AND EARLIER APPROACHES
  • HOMOLOGOUS AND NONHOMOLOGOUS REPAIR METHODS USED FOR NUCLEASE-BASED GENOME EDITING
  • POTENTIAL HUMAN APPLICATIONS OF SOMATIC CELL GENOME EDITING
  • SCIENTIFIC AND TECHNICAL CONSIDERATIONS ASSOCIATED WITH THE DESIGN AND APPLICATION OF GENOME-EDITING STRATEGIES
  • ETHICAL AND REGULATORY ISSUES POSED BY SOMATIC CELL GENOME EDITING
  • CONCLUSIONS AND RECOMMENDATIONS
• 5. Heritable Genome Editing
  • POTENTIAL APPLICATIONS AND ALTERNATIVES
  • SCIENTIFIC AND TECHNICAL ISSUES
  • ETHICS AND REGULATION OF EDITING THE GERMLINE TO CORRECT DISEASE-CAUSING TRAITS
  • REGULATION
  • CONCLUSIONS AND RECOMMENDATION
• 6. Enhancement
  • HUMAN GENETIC VARIATION AND DEFINING “NORMAL” AND “NATURAL”
  • UNDERSTANDING PUBLIC ATTITUDES TOWARD ENHANCEMENT
  • DRAWING LINES: THERAPY VERSUS ENHANCEMENT
  • HERITABLE GENOME EDITING AND ENHANCEMENT
  • CONCLUSIONS AND RECOMMENDATIONS
• 7. Public Engagement
  • PUBLIC ENGAGEMENT: BROAD CONCEPTS
  • U.S. PRACTICES
  • INTERNATIONAL PRACTICES
  • LESSONS LEARNED FROM PUBLIC ENGAGEMENT ACTIVITIES
  • MOVING FORWARD
  • CONCLUSIONS AND RECOMMENDATIONS
• 8. Summary of Principles and Recommendations
  • OVERARCHING PRINCIPLES FOR GOVERNANCE OF HUMAN GENOME EDITING
  • EXISTING U.S. OVERSIGHT MECHANISMS FOR HUMAN GENOME EDITING
• References
• APPENDIXES
  • A. The Basic Science of Genome Editing
  • B. International Research Oversight and Regulations
  • C. Data Sources and Methods
  • D. Committee Member Biographies
  • E. Glossary

Suggested citation:

National Academies of Sciences, Engineering, and Medicine. 2017. Human Genome Editing: Science, Ethics, and Governance. Washington, DC: The National Academies Press. doi: https://doi.org/10.17226/24623.