Next up for CRISPR: Gene editing for the masses?

The Genie is out of the bottle, and no one will be able to put it back in!


How CRISPR therapy could cure everything from cancer to infertility

The imminent approval of the world’s first CRISPR treatment for sickle cell disease is just the start: soon this gene-editing tool could be used to tackle everything from cancer to high cholesterol and infertility

CRISPR gene editing exploded onto the scene in 2012. It came about thanks to the discovery that many bacteria produce so-called CRISPR-Cas proteins that can cut DNA at specific sites. The DNA-cutting part wasn’t new – biologists had already found proteins that can do this, but each protein could only bind to and cut DNA at one specific sequence. To target a different sequence, biologists had to alter the shape of the DNA-binding part of the protein, a difficult and expensive process taking many months.

The revolutionary thing about CRISPR-Cas proteins is that the target sequence is determined by a piece of “guide RNA” that links up with the Cas protein and binds to any matching DNA sequence – and RNAs are cheap and easy to make.

Within months, hundreds of labs worldwide were trying CRISPR gene editing in all kinds of organisms. Many studies involve using CRISPR for research: detecting viruses, say, or recording cells’ activities. But it is no longer restricted to labs. In some countries, people are already eating CRISPR-edited plants and animals – and in 2018, it was announced that three genetically modified children had been born in China. Their genes were edited using CRISPR technology at the embryonic stage by scientist He Jiankui in an effort to prevent them contracting HIV from their fathers (see “Editing eggs and sperm”). Jiankui was jailed for his actions.

Most remarkably, more than 75 clinical trials involving CRISPR are under way or have already been completed. This is a phenomenal achievement as new medical technologies typically take several decades to develop to the point where they are ready to try on people.

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