August 14, 2017
By Josh Baxt, BioSpace.com Breaking News Staff
In the biology community, CRISPR is like the latest boy band, generating fierce disagreements and the occasional swoon. The gene editing technique is dominating many conferences and has generated headlines for its potential to create “designer babies.”
Headline writers have been working overtime the last couple of weeks after a team of scientists in Oregon and San Diego modified the DNA of human embryos. Fortunately, those scary headlines are closer to science fiction than science fact.
The reality is that CRISPR and similar gene editing technologies are giving researchers new tools to interrogate biology – and that’s the real story.
This was evident at the Future of Genomic Medicine (FOGM) conference in March, during which CRISPR was often front and center. The Broad Institute’s Feng Zhang, who had recently won a patent suit filed by Jennifer Doudna and UC Berkeley, was an early speaker. Doudna was invited to the conference but could not attend. Fortunately, the focus was less on the legal drama than on the technology’s promise – though granted, the controversy was bubbling just under the surface.
Zhang’s talk centered on using a CRISPR technology called C2c2 to diagnose pathogens. He noted there may be a long list of CRISPR effectors, like C2c2, that have yet to be discovered. New techniques could lead to even more precise editing.
Zhang was immediately followed by Bill Lundberg, chief scientific officer at CRISPR Therapeutics. Lundberg focused on the company’s preclinical work on sickle cell and beta-thalassemia therapies. He covered the challenges of editing somatic cells, the advantages of adding protective genes rather than editing existing mutations and ongoing concerns about off-target effects.
Our brains tend to gravitate towards controversy: the germline editing and courtroom throw-downs, but the real news here is the science – slow, incremental, plodding science. CRISPR is not even ready for clinical use, but it’s getting there.
Which leads to the second half of Lundberg’s presentation: How do we ethically rollout gene editing? The National Academy of Sciences and National Academy of Medicine recently released a comprehensive report: Human Genome Editing: Science, Ethics and Governance. The report recapitulates the issues associated with gene editing and provides a framework for future oversight.
In particular, the paper offers some nuance on germline therapies, focusing heavily on medical need. Editing for height, hair color, smarts or athletic prowess are hardly necessities. On the other hand, removing a Huntington’s disease mutation could measurably reduce suffering for a family and its descendants. Still, even well-intentioned and ethically sound applications can raise questions. How will people with disabilities be perceived if germline editing becomes widespread?
The report offers an array of thoughtful criteria for therapeutic germline editing, and “recommends that genome editing for purposes other than treatment or prevention of disease and disability should not proceed at this time.”
The controversies surrounding gene editing are not going away. Later at FOGM, Michael Eisen from UC Berkeley circled back to the intellectual property issue, wondering aloud whether patenting technologies like CRISPR deters scientific progress.
Eisen believes the problem stems from the 1980 Bayh/Dole Act, which allows universities to patent government-funded research. According to Eisen, experiments are performed and publications delayed to advance patent claims, not science. The question of ownership distorts inquiry and discourages innovation. Eisen is running for the U.S. Senate, so perhaps he will have an opportunity to amend and/or replace Bayh/Dole.
The important thing with CRISPR, or any groundbreaking technology, is that science and policy walk forward together. In both realms, good practice means questioning our assumptions and following the path defined by the best evidence.
Josh Baxt has been a science and healthcare writer for more than 18 years, working at Scripps Health and the Sanford-Burnham Medical Research Institute before going freelance in 2011. He writes about molecular biology, genomics, pharmaceuticals, emerging medical technologies, regulation and public policy. He is based in San Diego.
