CRISPR/Cas9
Welcome and come learn about CRISPR/Cas9, a growing genome editing tool! This website was designed to help explain the science behind CRISPR/Cas9 as well as the policies regarding its use. Feel free to explore the pages that delve into its history and opinions from highly respected individuals. Enjoy!
Professor's Opinions
Dr. Francesca Storici
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Professor of Biology at Georgia Tech
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You need to look at case by case situations. Analyze all situations separately and avoid making generalizations.
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Need to ensure that it's completely safe.
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Embryonic cells should require much more regulation. You need to be very clear in your purpose with gene line editing.
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The future is growing exponentially with CRISPR/Cas9. It is becoming more accurate, with more applications and experts. It's not perfect, but improving daily.
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Ideally, policies would be global. But it's understandable that this isn't the case due to different cultures and values.
Dr. Gregory Gibson
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Professor of Biology at Georgia Tech
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Supportive of somatic CRISPR research, but not germline.
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Accidents can happen and may be dangerous. These can result in mutations to the researcher.
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We have strict regulations, and this is appropriate. We need to allow time for society to catch up.
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Sees potential in cell-based therapy.
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Think outside of the box for drug-based treatment.
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Still need to know more about the consequences of reintroducing cells back into the body.
Legislator's Opinions
We met with two different legislators to gauge their opinions on policies. We showed them a powerpoint outlining the science of CRISPR/Cas9, followed with questions, to which the answers are shown below.
Martha Zoller
State Policy Director for the Office of Senator David Perdue
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Embryos should be respected because they have the potential to become human. Unused embryos can even be adopted.
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It is difficult to build a universal standard that can be applied to the whole world, but partnering up with countries with cultures similar to ours is possible.
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Funding would ideally be private as opposed to federal. This ultimately comes down to how taxpayers feel.
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There should be fewer constraints from governmental regulations so that researchers can proceed in multiple paths without reapplying for funding.
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In our opinion: She appears supportive of research. The opinions presented appeared more geared towards general genetic editing as opposed to specifically CRISPR/Cas9.
Ryan Diffley
Legislative Assistant for District at the U.S. House of Representatives
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When you change the germline, you can't predict changes later on. This can potentially put you at risk for something else, and we have to protect the future generation.
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It is difficult to have universal policies for countries.
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He doesn't see there being much change in genetic editing policies now or in the future.
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The current bans will be kept in place for the forseeable future.
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Many people in the country are not as literate in scientific matters, and the pros and cons are not going to be understood by the vast majority.
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You need the public to back the government for successful policy change.
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Communication between scientists and the public is important. The average person needs to be able to understand, especially when using taxpayers dollars for funding.
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CRISPR/Cas9 seems more specific than previous genetic editing. If it can be perfected, it's differentiated from the rest.
White Paper
The Science Behind it All
Short for Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-Associated Protein 9
It's growing in applications!
History
What has already been accomplished?
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Correction of the heterozygous MYBPC3 mutation (causes hypertrophic cardiomyopathy) in human preimplantation embryos (Ma, 2017)
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Correction of dominant mutation in Crygc gene (causes cataracts) in mice
The U.S. vs. International Countries
U.S. Policies
Other Countries
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Nearly 60% of all worldwide trials have occurred in the U.S.
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Research is regulated by the local Institutional Biosafety Committees (IBCs)
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Federal regulation comes from the Clinical Laboratory Improvement Amendments
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NIH funding requires further regulations
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Embryonic research has additional regulations, and federal funding is unavailable
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NIH considers proposals for somatic cell gene transfer, but not embryonic
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Japan: Tries to assess the risk of intervention and regulate accordingly
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Latin America: Limits interventions to treat or prevent disease, not for enhancement
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European Union: EMA regulates medicinal use of gene therapy. Clinical research is nationally regulated.
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China: Laws don't address genome editing specifically, but it is rested via guidelines and suggestions.
Araki, M., & Ishii, T. (2014). International regulatory landscape and integration of corrective genome editing into in vitro fertilization. Reproductive Biology and Endocrinology,12(1), 108. doi:10.1186/1477-7827-12-108
As far as research is concerned, the current method of localized regulations is preferred. research into genome editing technologies is primarily regulated by oversight from institutional review boards and biosafety committees. Nationally, the Centers for Medicare & Medical Service (CMS) have additional regulations called the Clinical Laboratory Improvement Amendments (CLIA). These are primarily focused on ensuring that test results on human derived tissues are analytically valid. Otherwise, there are general guidelines from the National Institute of Health (NIH) and the National Academies of Science (NAS), and, although these regulations are not binding, if funding is received from other sources, IBCs generally focus on abiding by these suggestions, including. If embryos are used, additional guidelines are involved, and federal funding is generally prohibited. When moving on to therapeutic trials, the FDA regulates trials and approval similarly to gene therapies, some of which have undergone FDA trials.
Rather than approving research at an institutional level, there could be approval at a national scale or on an international scale. Such oversight could come from more specific, binding regulations from governing bodies such as the federal government or United Nations. Employing such a strategy could create more uniformity in what is and is not allowed. However, it could also slow down approval for research. Additionally, the issue of sovereignty makes any international solution unfeasible.
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To ensure the continued development of gene editing technologies, we recommend both fewer barriers to research and a clearer, more specific regulatory pathway for approving new gene editing technologies. Using CRISPR/Cas9 allows for more accurate, narrowly tailored modifications to the genome. Additionally, the availability of CRISPR has allowed many more research facilities to now engage in genome related experiments. This increases the need for laws specifically addressing research of genome editing. Currently, the FDA addresses Genome editing technology under the same regulations as gene therapy. If the FDA instead had a specific criterion for genome editing that is separate from the gene therapy, it would be better suited for allowing the development of gene editing technologies by providing a clearer, specifically tailored pathway for approval.
Our last recommendation is for regulations related to ethical considerations to be developed before these technologies become legal. This includes financial barriers to access. Current technology is still very expensive, and if it stays that way, the use and distribution of genome editing can be limited only to those who can afford it. This isn’t necessarily unique to gene editing technology. However, what is unique is the consideration that the technology could also be used for enhancement rather than just restoration. While limiting the application of this technology to only restorative applications was considered the more ethical option, this would invariably lead to more subjective, case by case assessments.