Not too long ago, ‘designer babies,’ genome editing and gene therapy were futuristic and expensive ideas that were available only to the most affluent elite.
Yet, today, this baffling feat is becoming shockingly affordable. In the last few years you may have been hearing about the new technology known as CRISPR/cas9, more commonly referred to as CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats). It is a new, precise, and efficient method for editing DNA.
Since its initial debut in 2012, CRISPR’s capabilities have leapt forward every year. Some highlights include modifications of genomes in non-viable human embryos by Chinese scientists, modifications in pig embryos by US scientists in an effort to create pig organs viable for human organ transplants, and more recently, the authorization for UK scientists to begin experimentation in modifying viable human embryos.
CRISPR/Cas9 is not without its drawbacks. Although its precision has improved since its first introduction, the risk of off-target cuts exists and can range from 0.1 per cent to more than 60 per cent, depending on the target cell and sequence used in the experiment. Its power to wipe out entire species in under a year can be disastrous and have widespread effects on entire ecosystems.
For example, scientists have shown that by mutating a single gene in a single mosquito to render it sterile, they could essentially kill off all mosquitos in roughly 12 generations (36 weeks). But what does that mean for bats who rely on mosquitos as a food source? Use in human embryos could lead to ‘designer babies’ with the ability to pick and choose certain traits, consequently reducing genetic variation in populations.
It is a rather inexpensive and simple technique that can be easily accessed, and dangerous if not regulated. There has been such an explosive reaction to this new technique that it has outpaced our ability to create legal and ethical guidelines, and mandates for its use.
CRISPR has opened up new avenues for researching genetic diseases and gene therapy. While there are a number of concerns that accompany this research, the potential advantages are notable. CRISPR is more precise, efficient, and affordable than past methods of gene editing. There is still a lot of work to be done but initial studies have shown promising results.
In Toronto, CRISPR gene editing is currently being tested as a way to prevent and eliminate hereditary diseases like cystic fibrosis or muscular dystrophy, which do not currently have cures. It also has the potential to regulate genes instead of simply editing them. This means that it could activate some genes and silence others (like those which fuel cancer growth).
CRISPR technology is propelling genetics research forward. There are a number of ethical concerns accompanying this advancement, and regulatory bodies need to catch up. As the leading agency responsible for regulating health products, Health Canada’s responsibility for creating a comprehensive regulatory framework will ultimately determine how CRISPR’s capabilities will affect our society.
The agency has prioritized a sub-program for Biologics and Radiopharmaceuticals for 2015-2016 (which covers gene therapy products) with the goal of generating a regulatory framework to develop, maintain, and implement the program. As the CRISPR technology is moving forward rapidly in Canada and abroad, this regulatory framework is urgently needed.
Health Canada must ensure that the regulatory framework adequately addresses the concerns that are surfacing with the development of CRISPR, both domestically and internationally. Harmonization of CRISPR regulations and requirements will be necessary to ensure equal protection for citizens and reduce medical tourism. Countries are moving forward at different paces — the United Kingdom for example has already approved use of CRISPR for research on human embryos.
In December 2015, the National Academy of Sciences (NAS) held an international summit on Human Gene Editing after the Chinese Academy of Scientists asked for a ban on clinical use of human germ line editing. The meeting concluded that research should continue with proper oversight, and that editing germ line cells should not proceed until safety and efficacy concerns have been resolved. These discussions should continue to form the basis of a comprehensive regulatory framework for the use of CRISPR worldwide.
So why does CRISPR matter to us as students? CRISPR is not elusive technology. It is already being used in many labs at the university because of its broad range of applications and its ability to rapidly create animal models for testing. Students have an obligation to understand the benefits of CRISPR technology and the current debates surrounding its application.
Students have an important voice to advocate for how CRISPR technology can be used in the future because its implications will directly affect our generation. CRISPR offers substantial benefits and holds promise as an inexpensive treatment for a variety of genetic disorders. However, its powers should be used cautiously so as to not let it become a destructive villain.
Anna Foster and Parmida Jafari are on the advocacy sub-committee of IMAGINE at U of T, a student-run community health initiative aimed at promoting and discussing healthcare in Toronto.