97 N.C. L. REV. 1041 (2019)
This Symposium on Legal, Ethical, and Policy Implications of New Gene-Editing Technologies was motivated by recent scientific developments in the field of gene editing. For years, genomic medicine has been hailed as the future of clinical treatment. The general premise is that doctors will use detailed information about a particular patient’s DNA (and other “biomarkers”) to custom-tailor diagnoses, advice, drug choices and doses, and other specifics of treatment. President Obama’s highly publicized Precision Medicine Initiative (now rebranded—cryptically—as the “All of Us” Research Program) illustrates both the hope and the hype.
Despite this hope and hype, genomic medicine has thus far had a limited effect on the day-to-day practice of medicine, and that effect has been most notable in cancer treatment (for example, the use of BRCA gene testing in treating breast cancer made famous by Angelina Jolie). The limiting factors have included the facts that (1) genes tend to influence the probability of getting a disease but rarely “cause” a disease in a deterministic sense; (2) the relative influences of environment, lifestyle, and epigenetic factors (changes in DNA’s immediate chemical environment in the body) on the ways genes are expressed are only beginning to be understood; and (3) for the rare cases of clear genetic-disease causation, treatment can only be symptomatic, since we have no “cures” at the genetic level.
In fact, the holy grail of genomic medicine has always been the ability not just to identify dangerous gene mutations but to fix them: to go into a patient’s cells and change a dangerous DNA sequence to a healthy one. There have been efforts to do “gene therapy” by using viruses and other vectors to add desired DNA into the patient’s cells. There have been some limited successes but also some catastrophic failures, most infamously the death of a teenage boy in Pennsylvania and cases of leukemia-like side effects in France. In hindsight, the problems were probably due to insufficient knowledge about the DNA-delivery mechanisms.
Now a new “gene-editing” technology, called CRISPR (or CRISPR-Cas9), may have the potential to provide a safe and effective way to cut out mutated sequences of DNA and paste in normal variants. As is so often the case in science, it is actually a new application of old knowledge—in this case, about the immune systems of bacteria. There is a long way to go before CRISPR becomes part of patient care, but, for the first time, there seems to be a way to leapfrog the use of potentially risky vectors to deliver DNA into a patient’s cells. The promise and potential value of the technology is reflected in the epic struggle underway over the foundational patent rights, featuring MIT and the Broad Institute on one side and the University of California-Berkeley and several European luminaries on the other—a biomedical Clash of Titans. Meanwhile, in 2015 a Chinese research team reported the first successful gene-editing intervention in nonviable human embryos, followed last year by a Chinese scientist’s claim to have edited the genome of twin baby girls.
The rapid development of CRISPR technology—in particular, the ethically dubious Chinese activities—has spurred consternation, debate, and governance proposals among scientists, bioethicists, lawmakers, and regulators. The contributors to this Symposium are all significant contributors to this emerging discourse. In this Symposium, our contributors explain gene-editing technology and explore its significant implications for law, ethics, regulation, and health policy from their varied perspectives. In this Introduction, I will give a brief, “CRISPR for Lawyers” overview of the technology and then provide a synopsis of each of the contributions to this Symposium.