Finns correct a congenital disease with genetic scissors – “genetic mayonnaise” as an aid

The Finnish tradition is enriched with arginine, a disease known as succinic aciduria or ASA-urea. It is a severe metabolic disease in which the body does not process proteins normally, but they produce a very dangerous accumulation of ASA and ammonia. Excess ammonia leads to impaired consciousness, coma and even death.

In Finland, newborns are screened for possible ASA cleft so that the risk of disease can be determined before symptoms develop. The treatment of the disease is a very precise diet for life and in severe cases a liver transplant.

Researchers from the University of Helsinki and HUS have succeeded in correcting the ASA-urea gene deficiency in cell cultures and demonstrated that ASA-uria can improve the disease's harmful metabolism.

The researchers engineered stem cells from the skin cells of ASA patients. The researchers then used the CRISPR-Cas9 method, or so-called gene scissors, to correct disease-causing genetic errors in stem cells. Finally, the researchers instructed the repaired stem cells to differentiate into liver cells, so they could see that the liver disease had indeed been cured and that the repaired cells no longer produced harmful ASA.

“In our research, we have shown for the first time that the genetic error in the ASA groove can be corrected using gene scissors without side effects in the cells. The genetically modified cells are also metabolically improved,” says a stem cell biologist at the University of Helsinki, an expert in genetic medicine. Kirmo Wardiowara Bulletin.

The study was published in Genetics In American Journal of Human Genetics.

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mRNA:

In the study, the researchers used mRNA packed into a lipid, or fatty membrane, to get genetic shear inside cultured cells.

“This “genetic mayonnaise” we have prepared is based on a recipe for a medical product already in use, which will make it easier for patients to use it in the future. Our next goal is to improve ASA life in mice,” says the doctoral researcher. Timo Geskinen from the University of Helsinki.

“The same gene editing method works in living animals and humans, but we still don't know how safe it is. That's why the subject needs to be studied first with experimental animals”, Keskinen continues.

Only some hereditary diseases have a cure. Even less often, permanent improvement is achieved through treatments.

“However, a permanent cure is possible if the disease-causing genetic error is completely removed. Thanks to basic research into genetic scissoring and other factors that precisely edit DNA, permanent repairs are gradually beginning to emerge,” says Vardiovara.

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