The release of genes can treat the damage caused by kidney disease “irreversible”

It was believed that the damage to the body caused by the most common types of kidney disease inherited. But now, animal studies indicate that correction of responsible mutations by editing CRISPR genes can reflect at least some of them.

PKD causes extensive changes to the kidneys over time. “It seems that it is unlikely to change even if you are correct [the mutation]Michael Kaminski of the University of Carrett – Berlin University at Medicine in Germany says. “But it seems that the disease may be more than plastic more than previously estimated.”

PKD leads to liquids that grow larger over time, and they are formed in the kidneys and often in the liver. These organs eventually fail, which means that people need dialysis or planting to survive. But even before the kidney failure, its damaged and lush condition can cause many other issues, from high blood pressure and infections to excessive pressure on other organs.

With the form of adults of the disease, which is It is estimated that it affects 12 million people all over the worldBig cysts may not grow enough to cause symptoms until people are in the thirties of life, and during this period there could be large -scale changes to the kidneys and liver.

Now, the Kaminski team used a form of CRISPR forms called the base editing to correct a boom in a gene called PKD1 Which causes the disease in the mice.

The methods used by his team mean that the mutant gene was mainly corrected in the liver, and the number and size of the cysts have decreased after treatment. Camensky says there are also signs of kidney improvement.

Separately, Xiaogang liS team in Mayo Clinic in Rothschest, Minnesota, A similar study did Using methods that target the kidneys better. This indicates that the number and size of the cysts can also be reduced, he tells me.

Both groups of researchers were used viruses to provide the mechanism of genes. This may be a problem if frequent doses are needed, because the immune response to viruses can prevent treatments from working. “This is a source of anxiety,” he tells me. “But so far, based on the animal model, the immune response is very limited.”

Camensky says that replacing viruses with fatty nanoparticles such as those used in flexible vaccines that will avoid the potential immunity problem, but these particles cannot penetrate deeply in the kidneys through the blood to be effective. “But I think the approach you will provide [lipid nanoparticles] Through the urethra can be more realistic. “

Another problem is that the basic liberation can only correct mono mutations and will not work with people whose illness is caused by longer mutations. But Lee says he has achieved similar results with a technique called the main editing, which can correct long mutations.

This work will be published in a scientific journal soon, he says, then aims to move to the tests in people. “When our paper is spread, I will try to organize a small clinical trial,” he says.

On a wider scale, the result of the team should inspire that PKD is vice versa, more research in this approach. Nowadays, the only approved treatment is a drug called Tolvaptan, which slows the progress and requires people to drink large quantities of fluids.

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