The Gene-Editing Revolution: What Is CRISPR-Cas9?

Not even five years ago, researchers came across a feature of bacterial genomes that has the potential to change the world in profound ways. CRISP-Cas9, as the resulting technology is known, enables scientists to program what amounts to a molecular pair of scissors to snip a piece of undesirable DNA and replace it with a different strand. The precision with which this can be accomplished, while anything but perfect, is unprecedented.

That may sound like the kind of thing that could cure horrible diseases and genetic disorders, increase the yield and quality of crops, and de-fang disease-carrying pests. Indeed, in the short time CRISPR has been with us, researchers have looked into all of these applications and more. Sticking points and remain, not to mention profound ethical questions, but one thing is sure: you’ll be hearing a lot more about CRISPR before too long.

How Does It Work?
CRISPR stands for “clustered, regularly interspaced short palindromic repeats.” It is a quality of some bacterial DNA that enables the bugs to create RNA – a sort of single-strand pseudo-DNA – that seeks out DNA matching its sequence of “bases.” The bacteria will then send Cas9, a protein, to attack the marked DNA (recent research suggests another protein, Cpf1, may work as well or better). For bacteria, the targets are viruses. With a bit of tinkering, CRISPR-Cas9 can also be used to replace the targeted DNA with something more preferable.

This video explainer is courtesy of Intellia Therapeutics.

What Can You Do With It?
In human hands, this ability amounts to what’s known in common parlance as playing God. It offers an enormous improvement on existing gene therapies: researchers have played around with treatments for HIV, Huntington’s, hemophilia, cancer and horrific rare diseases such as Sanfillipo. It has agricultural applications that would make Monsanto Co.’s (MON) mouth water (more on that below). It also offers the ability, in theory, to do incredibly useful things like shut down the ability of mosquitoes to transmit diseases.

Researchers have used CRISPR on macaques, sorghum, zebra fish, rice and other species. They’ve treated liver disease in mice. A Chinese team has used CRISPR to edit a non-viable human embryo.

What Should You Do With It?
That’s where the ethical issues get sticky. CRISPR-Cas9 could in theory be used to “improve” human beings, making them faster, smarter or taller, not just less prone to genetic disorders. Many find this repugnant on its face for philosophical reasons. The practical implications are also sobering. Since only rich people are likely to be able to afford such treatment, will we be faced with a society in which economic inequality is compounded by biological inequality?

That brings up the specter of eugenics. Will CRISPR be used to “improve” people in ways that are blatantly subjective, by giving kids the “right” eye color, or worse? It could probably be harnessed to exploit people in terrible ways, too.

A large bloc of scientists opposes any modifications to “germ line” cells, those that will pass their genetic material onto the next generation. After all, even if every one is ethically squeaky-clean about their use of CRISPR, what if they make an irreversible, potentially multigenerational mistake?

The National Academy of Sciences met earlier this month to discuss the implications of CRISPR and the guidelines that should apply to its use. They are expected to issue a report later this year.

Who Owns It?
Monsanto might be interested in CRISPR, but it’s not wild about the legal battle raging over the intellectual property rights, and has stated that it will keep its distance until the issue is cleared up. Two scientists, Feng Zhang of MIT’s Broad Institute and Jennifer Doudna of Berkeley, are fighting over CRISPR’s central patent. Zhang is the “junior party” in the dispute, meaning that the burden of proof falls on him.

The decision could have implications for Editas Medicine Inc. (EDIT), a company that develops CRISPR therapies and is one of only four U.S. IPOs this year. The company’s website lists both Dr. Zhang and Dr. Doudna as founders, but Doudna is no longer involved with the company and has licensed her patent to Intellia Therapeutics. Zhang has licensed his patent to Editas. Doudna’s Caribou Therapeutics, another CRISPR startup, was recently awarded a different patent, for using poteins besides Cas9 in conjunction with CRISPR.

The Bottom Line
CRISPR has enormous potential to improve people’s lives as well as significant potential to make them worse. Questions remain, not least about who has the intellectual property rights to the technology. If anything’s certain, it’s that people will be talking more and more about CRISPR, so it pays to be ahead of the curve.

Read more: The Gene-Editing Revolution: What Is CRISPR-Cas9? | Investopedia


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