This report describes a gene editing technology, known as CRISPR-Cas9, with the potential to revolutionize genetic engineering and the biotechnology industry. CRISPR-Cas9 is a gene editing technology that offers the potential for substantial improvement over other gene editing technologies in ease of use, speed, efficacy, and cost. Many in the scientific, engineering, and business communities believe that CRISPR-Cas9 may offer revolutionary advances in the investigation, prevention, and treatment of diseases; understanding of gene function; improving crop yields and developing new varieties; production of chemicals used in biofuels, adhesives, and fragrances; and control of invasive species. Federal R&D Funding and Scientific Publications

The potential of CRISPR-Cas9 gene editing is further reflected in the rapid increase in CRISPR-related federal research funding and scientific publications. The Coordinated Framework for the Regulation of Biotechnology

The fundamental federal guidance for regulating biotechnology products, including those developed using CRISPR-Cas9, is the Coordinated Framework for the Regulation of Biotechnology (the Coordinated Framework) originally published in 1986 by the White House Office of Science and Technology Policy (OSTP). To assess the future landscape of biotechnology products, EPA, FDA, and USDA commissioned a study in early 2016 by the National Academy of Sciences (NAS) to identify (1) major advances and potential new types of biotechnology products over the next 5 to 10 years, (2) potential future products that might pose a different type of risk relative to existing products and organisms, (3) areas in which the risks or lack of risk relating to biotechnology are well understood, and (4) the scientific capabilities, tools, and expertise that may be useful to the regulatory agencies as they oversee potential future products of biotechnology. Some sections include a description of issues for congressional consideration, such as the regulation of future biotechnology products, international implications, and societal, ethical, environmental, and national security concerns. Effective modification, reduction, or elimination of the Anopheles mosquito—the primary vector for the transmission of malaria—could substantially reduce these costs and open up new economic opportunities in many of the world's poorest nations. The concerns of scientists and others have been, to some extent, borne out by the recent unverified claim of the birth of children with CRISPR modified DNA. However, ethical concerns about the genetic modification of the human germline are not new. In addition, scientists have conducted some of the early research using CRISPR-Cas9 to modify non-viable embryos in an attempt to mitigate some of the ethical concerns with this type of research. The publication of the first study using CRISPR-Cas9 in human embryos prompted the debate over germline modification to re-emerge in the scientific community, intensified by the perception that the new technology may be promising in ways not previously seen with respect to its ease of use and precision of editing. However, this approach also introduces genes that are not wanted. Some scientists have called for the development of reversal or immunization gene drives as a means to counter any unintended consequences with the open environment release of a gene drive-modified organism. Besides the scientific questions of risk in making changes to complex and interwoven ecosystems, these examples raise a number of questions about the use of gene drives and what may be considered socially acceptable. However, it may be possible to use gene editing to circumvent current mitigation strategies.
Scientists have long sought the ability to control and modify DNA—the code of life. A gene editing technology known as CRISPR-Cas9 offers the potential for substantial improvement over other gene editing technologies in that it is simple to use and inexpensive and has a relatively high degree of precision and efficiency. These characteristics have led many in the scientific and business communities to assert that CRISPR-Cas9 will lead to groundbreaking advances in many fields, including agriculture, energy, ecosystem conservation, and the investigation, prevention, and treatment of diseases.
Over the next 5 to 10 years, the National Academy of Sciences projects a rapid increase in the scale, scope, complexity, and development rate of biotechnology products, many enabled by CRISPR-Cas9. Concomitant with the promise of potential benefits, such advances may pose new risks and raise ethical concerns. For example, a Chinese researcher recently claimed that he had created the first genetically engineered human babies. According to the researcher, he used CRISPR-Cas9 to disable a gene that will make it harder for the twin girls, who were born in November 2018, to contract human immunodeficiency virus (HIV). The as yet unsubstantiated claim has sparked outrage and ethical debates by the international scientific community and others. Prior use of CRISPR-Cas9 gene editing in human embryos was generally limited to non-viable embryos, in part, to address ethical concerns such as the fact that the genetic change would affect not only the immediate patient, but also future generations who would inherit the change.
Additionally, CRISPR-related approaches (gene drives) are being considered to reduce or eliminate the mosquito that serves as the primary vector for the transmission of Zika or malaria, thereby improving public health. Some scientists and environmental groups have raised ethical questions and expressed concerns about the unintended ecological consequences of eliminating a species or introducing a genetically modified organism into an open environment.
Some experts assert that the current system for regulating biotechnology products—the Coordinated Framework for the Regulation of Biotechnology—may be inadequate, with the potential to leave gaps in oversight. Regulatory gaps may lead to increased uncertainty that could affect the development of future biotechnology products or a loss of public confidence in the ability of regulators to ensure that such products are safe.
In the 116th Congress, policymakers may want to examine the potential benefits and risks associated with the use of CRISPR-Cas9 gene editing, including the ethical, social, and legal implications of CRISPR-related biotechnology products. Congress also may have a role to play with respect to regulation, research and development, and economic competitiveness associated with CRISPR-Cas9 gene editing and future biotechnology products.