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Revolutionary Gene Therapy Using Crispr-Cas: Dutch Researchers Make Medical Breakthrough

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NOS Nieuws•gisteren, 20:13

Thanks to a new gene therapy, ten patients, including two Dutch people, are suddenly almost completely free of the complaints of their hereditary swelling disease, it was announced today. “This is completely overwhelming,” said Danny Cohn, internist and researcher at Amsterdam UMC NOS Radio 1 News.

The mechanism behind this therapy is known as crispr-cas. What kind of therapy is this and why is it so special?

Crispr-cas is a precision technique that makes it possible to cut DNA, the building block of life, very accurately. This makes it possible to make adjustments to genes that cause diseases, such as the swelling disease in the new study. The technique is now also widely used in crops, for example to make them more resistant to drought or to produce more fruit.

Ancient mechanism

What is special is that the technology was not invented, but discovered. Bacteria have been using the mechanism for billions of years to eliminate viruses. You can compare this mechanism a bit with the way the police sometimes try to catch a criminal: by distributing photos of the criminal. If someone matches the photo, you go after that person.

Bacteria do this by storing small pieces of DNA from viruses in their own DNA – the ‘photo’ by which they can recognize the viruses. If they come across such a piece of DNA somewhere, they send molecular scissors to it to cut the DNA and thus eliminate the virus.

Scientists soon discovered that you can determine for yourself which piece of DNA the molecular scissors cut, simply by giving them the right ‘photo’: a small piece of the genetic code, for example of cystic fibrosis or swelling disease. This way you can make very precise changes in the DNA, down to gene accuracy.

Fast Nobel Prize

That discovery earned Jennifer Doudna and Emmanuelle Charpentier the 2020 Nobel Prize in Chemistry, just eight years after their discovery. Most scientists have to wait decades before their research is rewarded with the coveted prize, but insiders thought eight years would still be a long time for crispr: “The time has finally come,” said microbiologist John van der Oost of Wageningen University & Research in 2020.

What does the abbreviation crispr-cas stand for?

Crispr is an abbreviation that stands for Clustered Regularly Interspaced Short Palindromic Repeats. They are small pieces of DNA in the genome of bacteria that are repeated over and over again.

Cas (Crispr ASsociated proteins) is a category of proteins that unzip DNA and then cut it in half. They are a kind of molecular scissors. They come in different variants, each with its own number. Cas9 is the best known.

In the meantime, the technology is being made more and more precise, the so-called prime editing. This no longer involves cutting an entire gene, but allows you to very accurately adjust the building blocks of a single gene. This promises even fewer side effects.

Not everyone is just enthusiastic about the new technology. There are concerns that it may lead to unwanted side effects in humans or that large companies want to use the technology to gain more power. Partly for these reasons, the European Union was until recently reluctant to allow the use of CRISPR-cas, but has now broadened its policy.

Suicide to survive

Meanwhile, scientists are still discovering new variants of CRISPR systems. Researchers from Wageningen University & Research are publishing a study in the scientific journal today Sciencewhich shows that a type of CRISPR system can sometimes trigger an entire chain reaction that has eluded scientists until now.

In some cases, the CRISPR mechanism not only disables the virus, researchers discovered to their surprise, but also the cell itself through a complicated process. The cell therefore commits suicide, as it were. Or better: the cell sacrifices itself for the group. “The mechanism kills the cell, which saves the population,” says microbiologist Raymond Staals, one of the researchers. “Altruism in Bacteria.”

The research into this is still in its infancy, but Staals sees opportunities to use it for cancer therapy. If you could build this mechanism into tumor cells, they could become suicidal and clear themselves away.

The possible applications of CRISPR-cas continue to expand, from agriculture and food to healthcare.

2024-02-01 19:13:21
#agriculture #healthcare #crisprcas #revolutionary

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