When news circulated last week about OHSU’s groundbreaking research in gene repair, I was as surprised as anyone, but not by the news itself. As senior vice president of research at OHSU, I was eagerly anticipating our ability to share the peer-reviewed work led by Shoukhrat Mitalipov and published Wednesday in Nature, one of the world’s foremost scientific publications.
Scientific journals rely on embargoes to ensure accurate and thoughtful reporting, including well-informed opposing views. To honor that embargo, OHSU agreed to hold off discussing these important findings until the paper was officially released. Unfortunately, word of these findings was revealed in several media outlets ahead of that release.
Now is the right time to welcome discussion about the many implications of this discovery.
Dr. Mitalipov and colleagues have successfully removed a lethal genetic defect in human embryos using the gene editing tool known as CRISPR. This breakthrough is profound. It is the initial confirmation that a life-threatening genetic defect can in theory be erased. The new technique targets a mutation in nuclear DNA that causes hypertrophic cardiomyopathy, a common genetic heart disease that can cause sudden cardiac death and heart failure. This new method repairs the mutation and prevents it from being inherited by succeeding generations.
Beyond this specific use, the research has proved essential in validating the legitimacy of continued exploration of gene-editing tools in the human embryo.
This basic science research was conducted under the strictest ethical oversight, with guidance from our Innovative Research Advisory Panel and reviews by the OHSU Institutional Review Board, Scientific Review Committee, and Data Safety Monitoring Committee.
This approach is also in concert with the report issued by the National Academies of Science earlier this year, which concludes that basic research in this area is essential to making wise decisions about using these techniques in humans.
Although Dr. Mitalipov’s research began before the report was published, we were encouraged that the National Academies unhesitatingly supports basic research in this arena and is largely supportive of gene editing that can’t be inherited. With respect to heritable editing of the genome, such as that published by Dr. Mitalipov today, it advocates a cautious approach.
Decisions about how and whether to use CRISPR or other gene-editing tools on people are ultimately questions society must address. The answers will require all of us to work out together their moral and ethical implications before we engage in clinical trials of this research. Public engagement is critical. And we start that conversation with the facts.
This viewpoint originally ran as a guest opinion piece in The Oregonian Monday, August 7, 2017. Daniel Dorsa, Ph.D., is senior vice president for research at Oregon Health & Science University in Portland.