News and opinion regarding CRISPR genomic alteration technology often veers into extremes; this will either be the greatest tool ever or will entice humanity to ruin. The prospect of immense financial rewards has induced the swift spawning of companies determined to exploit an extraordinary opportunity. Developments are coming at a fast pace.
CRISPR genetic modification could be the key to controlling some diseases and a general sense of urgency has already propelled the technology into clinical trials. However, questions as to whether researchers have gotten too far ahead of the data have been raised (1). A controversial new study suggesting CRISPR editing may produce large numbers of untargeted DNA alterations has been met with a ferocious push back from eminent scientists and companies involved in developing the new technology (2, 3). Disputing the methods and data and alleging faulty peer review, some have called for the journal to retract the paper. For the involved publically-traded companies subject to market forces the stakes are high. Stock valuations declined sharply in response to the publication of results highlighting the potential risks of CRISPR.
In one sense, the notion that CRISPR-Cas9 activity is imprecise is not controversial because investigators have been working for some time to improve the system (4). However, right or wrong, the new findings were attention-grabbing and forcing a retraction of that paper may help companies minimize financial losses. Although a retraction may satisfy pressing corporate concerns, it does not resolve the controversy surrounding off-target mutations generated by CRISPR therapy. One problem is that not much published data can be cited to establish whether or not competing claims about genomic editing manageability are reliable. This situation fuels a perception CRISPR technology is being rushed into the clinic based on sketchy information. Perhaps companies will now be motivated to fill this information gap by publishing studies which establish the targeting efficiency and error induction rates of their genetic alteration systems.
A troubling situation envisioned nearly 10 years ago by Nobel laureate Paul Berg may now have emerged (5). Where academic scientists could once be counted on to act as honest brokers regarding the risk and benefits of new technologies, the situation we face today is much more complicated. Many eminent scientists involved in CRISPR research and development have vested financial interests in the exploitation of this technology. I feel sorry for the journal editors who must now adjudicate the issues swirling around a published paper that has offended some powerful persons and organizations. At some point this particular dispute will be resolved. For the sake of those volunteering for tests of CRISPR-based therapies it is vital to establish the scientific details supporting the clinical use of this technology with rigor and precision.
(1) A. King. 2017. Fears that Gene-Editing Cancer Trails Are Premature. Chemistry World, 9 January 2017. https://www.chemistryworld.com/news/fears-that-gene-editing-cancer-trials-are-premature/2500206.article
(2) Antonio Regalado. Gene Editing Companies Hit Back at Paper That Criticized CRISPR. MIT Technology Review, 9 June 2017. https://www.technologyreview.com/s/608073/gene-editing-companies-hit-back-at-paper-that-criticized-crispr/?set=608061
(3) I. Haydon. 2017. CRISPR Controversy Raises Questions About Gene-Editing Technique. The Conversation, 31 May 2017. https://theconversation.com/crispr-controversy-raises-questions-about-gene-editing-technique-78638
(4) H. Ledford. 2016. Enzyme Tweak Boosts Precision of CRISPR Genome Edits. Nature, 6 January 2016. http://www.nature.com/news/enzyme-tweak-boosts-precision-of-crispr-genome-edits-1.19114
(5) P. Berg. 2008. Meetings that Changed the World; Asilomar 1975: DNA Modification Secured. Nature 455:290-291. http://www.nature.com/nature/journal/v455/n7211/full/455290a.html