The National Academies of Sciences, Engineering and Medicine

About the Study

Introductory Video

Alta Charo, one of the committee co-chairs, describes the committee’s task and why these issues are so important to society.

Gene-editing technologies hold great promise for advancing science and improving human health. Powerful new tools, such as CRISPR-Cas9, allow researchers with basic knowledge of molecular biology to precisely modify the genetic makeup of any living organism. The possible applications for such technologies are many.  In humans, the technologies could offer a cure to often devastating genetic diseases such as Huntington’s disease and sickle cell anemia, and help improve understanding and treatment of many other illnesses.

However, these new avenues of research also present many complex challenges, both to the scientific and medical communities and to society as a whole.  The availability of new technologies has intensified debate among scientists and physicians about such research.  We are at a critical juncture in genetic research.  What is needed now is guidance that is based on an in-depth review of the science underlying gene editing and an understanding of the potential benefits as well as the valid concerns raised by this research. 

Statement of Task

The study will examine the scientific underpinnings as well as the clinical, ethical, legal, and social implications of the use of human genome editing technologies in biomedical research and medicine.  It will address the following issues related to human gene editing, including editing of the human germline:

  1. What is the current state of the science of human gene editing, as well as possible future directions and challenges to further advances in this research? 
  2. What are the potential clinical applications that may hold promise for the treatment of human diseases?  What alternative approaches exist?
  3. What is known about the efficacy and risks of gene editing in humans, and what research might increase the specificity and efficacy of human gene editing while reducing risks?  Will further advances in gene editing introduce additional potential clinical applications while reducing concerns about patient safety?
  4. Can or should explicit scientific standards be established for quantifying off-target genome alterations and, if so, how should such standards be applied for use in the treatment of human diseases?
  5. Do current ethical and legal standards for human subjects research adequately address human gene editing, including germline editing?  What are the ethical, legal, and social implications of the use of current and projected gene-editing technologies in humans?
  6. What principles or frameworks might provide appropriate oversight for somatic and germline editing in humans? How might they help determine whether, and which applications of, gene editing in humans should or should not go forward? What safeguards should be in place to ensure proper conduct of gene-editing research and use of gene-editing techniques?
  7. Are there examples of how these issues are being addressed in the international context? What are the prospects for harmonizing policies? What can be learned from the approaches being applied in different jurisdictions? 

The committee will address these questions and prepare a report that contains its findings and recommendations. The report will provide a framework based on fundamental, underlying principles that may be adapted and adopted by any nation that is considering the development of guidelines. The report will also include a focus on advice for the United States. 


The sponsors for this study are the U.S. Food and Drug Administration, Greenwall Foundation, Wellcome Trust, MacArthur Foundation, and the Defense Advanced Research Projects Agency.


Katherine Bowman, Ph.D.
Study Director
Senior Program Officer
Board on Life Sciences
Division of Earth and Life Studies

Monica Gonzalez, M.P.H.
Associate Program Officer
Board on Health Sciences Policy
Health and Medicine Division

Joanna Roberts, M.P.P.
Senior Program Assistant
Board on Health Sciences Policy
Health and Medicine Division

Andrew Pope, Ph.D.
Director, Board on Health Sciences Policy
Health and Medicine Division

Fran Sharples, Ph.D.
Director, Board on Life Sciences
Division of Earth and Life Studies


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