Kendall Squared brings you dispatches from the world’s epicenter for biotechnology and drug discovery.
Hundreds of scientists are gathered in Washington D.C. this week to wrestle with the issues of if and how human genomes should be edited — a conversation that has far-reaching implications for researchers in Kendall Square.
The International Summit on Human Gene Editing is giving particular attention to the topic of editing genes in reproductive cells and embryos. That approach could prevent certain inherited diseases, but it remains controversial because the changes would be transmitted to future generations.
A panel on Wednesday highlighted another important role for such research: understanding more about the basics of human genetics and embryonic development.
Rudolf Jaenisch, a founding member of the Whitehead Institute for Biomedical Research and an MIT biologist, is one scientist invested in this kind of research. Jaenisch, who has studied embryonic stem cells, said at the summit he thought editing embryos’ DNA as a way of curing diseases would have limited effects for most genetic disorders at this point. But he argued that researchers could make great strides in understanding how humans develop by editing DNA in embryonic cells.
Studying animals like mice and monkeys can provide some insight, Jaenisch said, but “it’s important to really confirm that in human embryos.”
However, “to work with human embryos doesn’t mean we want to implant them,” he emphasized.
Gene editing in humans has garnered lots of attention lately because of a new technology called CRISPR that makes the process faster, easier, and more precise. Jaenisch said that his lab can use CRISPR to genetically alter mice in a matter of weeks when previously it could have taken two years.
The technology has been embraced by thousands of researchers, in local labs in Kendall Square and around the world. Companies, including Cambridge, Mass.-based Editas Medicine and CRISPR Therapeutics, are also trying to leverage the method to improve drug discovery.
One concern nagging CRISPR has been that, despite its improvements over other methods, it still leads to errors in the editing process. Researchers have been working to refine the tool, and on Tuesday, scientists at the Broad Institute, including CRISPR pioneer Feng Zhang, debuted their latest finding, a slightly tweaked version of the key enzyme that cuts down on what are known as “off-target effects.”
Labs around the world will have access to the enzyme, according to the Broad, which will further aid basic research. But at the same time, as the technology improves and CRISPR becomes safer to use in people, it seems likely that the ethical debate will only intensify.
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