Gene-editing regulations "urgently needed"
Scientists and policy makers are pushing for "urgent regulation" of gene editing technologies, in the face of ecological risks that are "real and poorly understood". Encoding genes to ensure all of a species end up male could be an attractive method of pest control, at least initially, but could have unknowable effects on the widedr ecosystem. We need to incentivise research into the risks associated with gene editing, with less than 1% of synthetic biology funding going to risk research. Currently, there is debate over which US governmental arm assumes responsibility for gene editing, with the FDA overseeing some aspects, and the Department of Agriculture others. Lastly, a public debate over the risks we are willing to take to further research and development is necessary. An interview with gene-editing technology pioneer, Jennifer Doudna, also in the New York Times argues that although sci-fi technologies such as embryo editing are realistically a long way off, we should still be ahead of the game in developing a regulatory framework to deal with the ethical quandary that may arise. Recalling a hypothetical discussion with peers, Mrs Doudna commented "There may come a time when, ethically, we can’t not do this."

Doudna arranged for a postdoctoral researcher, Martin Jinek, to collaborate with Charpentier’s team. After months of experimentation, they determined that Crispr relied on two separate kinds of RNA: a guide, which targeted the Cas9 protein to a particular location, and a tracer, which enabled the protein to cut the DNA. But even then, it wasn’t clear whether Crispr was anything more than a curiosity. Unlike most living things — people, animals, plants — the cells of bacteria have no nucleus, and their RNA and DNA interact in a different way. Because of that, Jinek says, it was hard to say ‘‘whether the system would be portable’’ — whether it would work in anything except bacteria. Going over the problem in Doudna’s office, Jinek began sketching the two RNA molecules on the whiteboard. In their natural form, the two are separate, but Doudna and Jinek believed that it would be possible to combine them into a single tool — one that was more likely to work in a wide range of organisms. ‘‘That was the moment the project went from being ‘This is cool, this is wonky’ to ‘Whoa, this could be transformative,’ ’’ Doudna says.

The tool Doudna ultimately created with her collaborators paired Crispr’s programmable guide RNA with a shortened tracer RNA. Used in combination, the system allowed researchers to target and excise any gene they wanted — or even edit out a single base pair within a gene. (When researchers want to add a gene, they can use Crispr to stitch it between the two cut ends.) Some researchers have compared Crispr to a word processor, capable of effortlessly editing a gene down to the level of a single letter.

Even more surprising was how easy the system was to use. To edit a gene, a scientist simply had to take a strand of guide RNA and include an ‘‘address’’: a short string of letters corresponding to a particular location on the gene. The process was so straightforward, one scientist told me, that a grad student could master it in an hour, and produce an edited gene within a couple of days. ‘‘In the past, it was a student’s entire Ph.D. thesis to change one gene,’’ says Bruce Conklin, a geneticist at the Gladstone Institutes in San Francisco. ‘‘Crispr just knocked that out of the park.’’

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Genetic engineering has wrought spectacular changes in the years since it was first developed in 1973. By breeding mice to have particular mutations, researchers have been able to explore the roots of diseases including cystic fibrosis and diabetes. It has also opened the door to new hybrids: pest-resistant corn with genes taken from bacteria, for instance, and yeast modified to churn out an antimalarial drug. As of 2014, the market for genetically engineered products was worth almost $2 billion — a number that is expected to double over the next five years.

But despite these advances, the process of altering genes has remained laborious and inexact. Engineering a mouse with a single mutation took a dedicated lab almost two years, and even that was something of a crapshoot. Altered genes frequently ended up in random locations, or else in widely varying numbers — no copies in one cell, a dozen copies in another — often with confounding results. One scientist told me that before Crispr, he had to microinject roughly a million cells in order to get one perfect mutation. With Crispr, he could get the same result using just 10 cells.