CRISPR offers new hope to treat diabetes

GENA CRISPR GENE TECHNOLOGY In recent years, he showed his revolutionary potential: used to treat rare diseasesto Customize crops to withstand the extremes of climate change or even Change the color Spider web. But the biggest hope is that This technology will help you find a medicine for a global disease, such as diabetes. New studies in that direction.

The researchers first managed to implant Crispr-decorated pancreat cells in man with type 1 diabetes, autoimmune diseases in which the immune system of attacking insulins in Pancreas. Without insulin, the body can then not regulate blood sugar. Unless undergoing steps for glucose level management by other means (usually, injecting insulin), this can damage nerve and organs – especially heart, kidneys and eyes. Approximately 9.5 million people around the world have type 1 diabetes.

In this experiment, changed to cells for months produced insulin after they implanted, without having to make any immunosuppressive drugs to stop their body to attack cells. CRISPR technology enabled researchers to autopsy genetically modified cells with camouflage to avoid discovering.

Study, published last month in New English magazine medicine, Details details ended. First, the pancreatic cells are left to the deciduous donor without diabetes, and then the CRISPR-CAS12B gene editing technique will allow them to avoid the patient’s immune response for diabetes. The cells change as it says “hypoimmune,” explains Sonja Schrepfer, Professor at the Medical Center of Cedars-Sinaya in California and Scientific Kofaunder Biotechnology Sana, a company developed by this treatment.

The decorated cells were then implanted into the patient’s forearm muscles, and after 12 weeks, the signs of rejection were not detected. (A Subsequent report From the biotechnology, Sana notes that the implanted cells continued to avoid the patient’s immune system after six months.)

The tests performed as part of the study were recorded that cells were functional: implanted cells excrete insulin in response to the level of glucose, represent key towards diabetes control without the need for insulin injections. Four unwanted events were recorded during monitoring with the patient, but none of them were serious or directly connected to modified cells.

The ultimate goal of the researcher is to apply immune-camouflage of genes to stem cells – which have the ability to play and differences in other cellular types inside the body – and then direct their development in the cells from the island insulin. “The advantage of an engineering registry stem cell is that when these stem cells are prolifered and created new cells, new cells are also hypoimune,” Schrepfer explained to A Cedars-Sinai K + A Earlier this year.

Traditionally, transplanting foreign cells in the patient needs to suppress the patient’s immune system to avoid refusing them. This carries significant risks: infections, toxicity and long-term complications. “See patients die from refusing or serious complications of immunosupsia and I decided to focus my career on the development of strategies without immune drugs,” Cedars-Sinai “Schrepfer-Sinai said.

Although the research marks the turning point in search of the treatment of type 1 diabetes, it is important that the study included the participant itself, which was not enough to be sufficiently to control the patient injected insulin. Editorial board in the journal Nature He also says that some independent research groups failed in their efforts to confirm that Sana method enables regulations to the ability to avoid the immune system.

Sana will ask to lead more clinical trials that start next year. Without overtime of criticism and restrictions on the current study, the possibility of transplanting cells modified that it is invisible for the immune system opens a very promising horizon in regenerative medicine.

This story originally appeared Wired Spanish and has been translated from Spanish.