Mutated animals show why gene editing isn’t ready for human trials

Big Mistake

The scientific community has largely denounced He Jiankui, the researcher who recently claimed to have used CRISPR to alter the genomes of twin babies in China.

A key critique has been that CRISPR’s effects aren’t understood well enough to guarantee the twins’ wellbeing. On Friday, The Wall Street Journal published a disturbing story about the unintended consequences CRISPR gene editing has had on animals, and it paints a portrait of a technology nowhere near ready for human trials.

Side Effects May Include

The WSJ article describes a number of examples of geneticists using CRISPR to modify livestock to exhibit favorable traits.

In each example the editing worked, but it also produced unintended side effects. Rabbits and pigs modified to grow leaner also grew enlarged tongues and extra vertebrae, respectively. Goats modified to grow longer cashmere wool grew too large in the womb for natural births, while cattle that scientists modified to sport lighter coats died as calves.

If we can’t yet prevent our CRISPR edits from producing these unintended consequences in animals, we certainly shouldn’t be editing the genes of human beings.

Cranked Up

But we might not need to edit genes for CRISPR to be useful. On Friday, researchers from the University of California San Francisco published a study in the journal Science in which they describe CRISPRa, a variation on CRISPR that doesn’t require any editing at all. Instead of cutting a targeted gene, this modified system amplifies the gene’s activity.

The most common genetic mutation in severely obese people is in the genes SIM1 or MC4R, both of which are essential for regulating hunger and satiety. For their study, the researchers genetically engineered mice to have a mutation in one of those genes. Then, they used CRISPRa to amplify the expression of the non-mutated gene. This allowed the mice to regulate their hunger as effectively as if they had no mutation at all.

Eventually, CRISPRa could allow us to address obesity and a host of other genetic disorders in humans — all without having to undertake the risk of editing a single gene.

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By Kristin Houser / Writer/Editor and Content Manager

From brainstorming story ideas to proofreading final copy, I am passionate about each step in the writing process, and for more than a decade, I have channeled that passion into creating (and helping others create) stellar content.

In addition to my position as an associate editor for Futurism, a science and technology publication that is currently read by over 30 million people monthly, I have contributed dozens of articles to iQ By Intel and am the managing editor of my own content-rich music website catering to the Los Angeles market.

When I'm not writing articles in Word or WordPress for those sites, you can find me tweaking dialogue in Final Draft, which I've used to write scripts for several produced web series and commercials, both animated and live action.

While the majority of my writing experience has been geared toward technology, entertainment, and education, I am always interested in opportunities to explore new arenas, so if you're looking for someone to contribute at any stage of the writing process, I'd love to hear from you.

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(Source: futurism.com; December 17, 2018; https://tinyurl.com/ydbtqqae)
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