Stephan Walter
鈥淭he pain was like being struck by lightning and hit by a freight train all at the same time,鈥 Victoria Gray聽told聽New 女生小视频聽in 2023.聽鈥淣ow everything is different for me.”
Gray used to experience severe episodes of sickle cell disease, but in 2019 she was effectively cured by a revolutionary technique that enables changes to be made to specific bits of our DNA: CRISPR gene editing. In 2023, that experimental treatment became the first approved CRISPR therapy.
There are hundreds of clinical trials of CRISPR-based treatments聽, and this is just the start. CRISPR could help treat all kinds of diseases, not just genetic聽conditions. For instance, a single dose of CRISPR could reduce your risk of heart attacks and strokes by聽permanently lowering your cholesterol levels.
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And while it isn鈥檛 yet safe enough to attempt, it does seem likely that in the future, CRISPR will be聽routinely聽used to alter our children’s genomes聽to reduce their聽risk of common diseases.
CRISPR is also starting聽to聽transform farming by making it much easier to develop crops and livestock that are disease-resistant, adapted to warmer conditions or聽better for聽eating.
Given all this, there is no doubt that CRISPR is one of the very best ideas of the 21st century. Its power lies in its ability to correct 鈥渟pelling mistakes鈥 in DNA. There are two parts to this: first, you have to get your gene-editing tool to the right place in the genome, like moving your cursor to the right spot in a long document on a computer. Next, you make the change.
Microbes use this mechanism in their battle with other microbes and,聽prior to 2012, biologists had discovered many natural gene-editing proteins. However,聽each one targeted just one location, or sequence, in the genome.聽To edit a different spot, the only聽option聽was to redesign the part of聽the聽protein that binds to DNA to target another sequence, a laborious process that聽took years.
But it turns out that bacteria have evolved a big family of gene-editing proteins that don’t bind to DNA directly. Instead, they hook up with a piece of RNA 鈥 a cousin of DNA 鈥 and search for sequences that match the RNA. And making RNA takes days, not years.
In 2012, Jennifer Doudna at the University of California, Berkeley, and her colleague at the Max Planck Institute for Infection Biology in Berlin showed how one of these gene-editing proteins, called CRISPR Cas9, could be 聽by adding the right form of “guide RNA”.
There are now thousands of variants of CRISPR being used for many purposes, but all rely on guide-RNA targeting. It is a world-changing technology, for which Doudna and Charpentier were聽awarded a Nobel prize聽in 2020.
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