Beyond-DNA Day
Happy DNA Day! Eleven years ago today the Human Genome Project came to a close. The years since have seen countless refinements of that initial understanding of the human genome and the roles that genes do and do not play in determining who we are. These years have also served to remind us of the complexity of the universe and our imperfect knowledge of its mechanisms – how is it possible that amoebas have 200 times more DNA than we do?! Genetic determinism may finally (slowly) be starting to be acknowledged as an archaic notion, an unscientific concept that’s been impossibly muddied by all the strange ways in which the world actually works.
But perhaps this DNA Day will be remembered not for our increased understanding of the human genome, but for our increased attempts to change it.
On April 15, the US Patent and Trade Office awarded its first patent for the components and methodology of CRISPR – the “powerful new way to edit DNA,” that allows “customizing the genome of any cell or any species at will.” There are still serious technical problems to work out, but there’s no denying that this is currently one of the hottest areas in biotech research. Feng Zhang of the Massachusetts Institute of Technology, one of the recognized leaders of the field and the listed inventor on the patent, was one of Nature’s 2013 “Ten people who mattered this year,” an MIT Technology Review Innovator Under 35 in 2013, and recently received The National Science Foundation’s Alan T. Waterman Award.
The patent was given to the Broad Institute of MIT and Harvard, but they may well choose not to enforce it too strictly. Many other organizations are currently using the patented methodology, including the startup Editas, which was co-founded by Zhang and hopes to utilize CRISPR to treat a broad range of genetic diseases. Another founder of Editas is Jennifer Doudna, who was actually the first to uncover the multiplicity of uses possible for the CRISPR system. She and the University of California have their own patent application pending, and it’s unclear if that can be granted now.
According to MIT Technology Review, “the Broad is keeping a tight lid on their plans for the patent,” but it would be surprising if they were to opt to retain the technology for their exclusive use. Broad Institute director Eric Lander has actively criticized the idea of using patents to restrict research, in an amicus brief in the Myriad case. Also, as a member of the Global Alliance, it would be strange if Broad opted for onerous restrictions; they may see the point of holding the patent as primarily avoiding abuse of the technology (though definitions of abuse may vary).
Genetic modification got another boost recently, as the FDA just granted its first approval of a gene therapy treatment. The company Celladon received breakthrough status for its treatment MYDICAR, which showed substantial promise in its Phase 1 trial for reducing heart failure in people lacking a sufficient amount of a particular enzyme. The death of Jesse Gelsinger in 1999 caused many to take a step back from gene therapies. But a string of recent articles have concluded that the technology is making a comeback and Celladon’s success seems to suggest that is the case.
Genome editing opens up many possibilities, but attempting to perfect what we imperfectly understand will surely lead to some unintended consequences. A new report from Tetsuya Ishii et al. in Cell titled “Caution required for handling genome editing technology” makes the case that new genome editing technologies show promise, but raise technical issues such as unforeseen toxicity, off-target mutagenesis, and mosaic modifications. The authors point out that some of the new technologies blur the boundaries of current regulations and they argue that this moment “may provide an important opportunity to form a new global consensus for future regulations in the field of genetic engineering.” Additionally, they note that “in order to achieve a better relationship between biotechnology and society, researchers must act with caution and establish a scientifically valid assessment method for evaluating organisms that have been modified with genome editing.”
As efforts to manipulate DNA ramp up, and creep into our bodies and the bodies of those we love, safeguards will be critical. The implementation of guidelines by next DNA Day would be an achievement worth celebrating.
Previously on Biopolitical Times: