CRISPR gene editing technology Its revolutionary potential has been proven in recent years: it has become accustomed to it Treat rare diseasesarrive Adapt to crops Tolerate the extremes of climate change, even Change the color Cobweb. But the biggest hope is This technology Will help find cures for global diseases, e.g. diabetes. A new study points out this direction.
For the first time, researchers have successfully implanted a man with type 1 diabetes, an autoimmune disease in which the immune system attacks insulin-producing cells in the pancreas. Without insulin, the body cannot regulate blood sugar. If no steps are taken to manage glucose levels by other means, usually by insulin injection, it can lead to damage to nerves and organs – especially the heart, kidneys and eyes. About 9.5 million people worldwide suffer from type 1 diabetes.
In this experiment, cells edited after implantation produced insulin several months without the recipient taking any immunosuppressive drugs to stop their body from attacking the cells’ bodies. CRISPR technology enables researchers to impart genetically modified cells to evade detection.
The study was published last month New England Journal of Medicine,,,,, Detailed description of the step-by-step process. First, islet cells were taken from the donors of the deceased who did not have diabetes, and then changed using gene editing technology CRISPR-CAS12B to enable them to escape the immune response of diabetic patients. The cells are called “low immunity,” explains Sonja Schrepfer, a professor at Cedars-Sinai Medical Center in California and a science co-founder of Sana Biotechnology.
The edited cells were then implanted into the patient’s forearm muscles, and after 12 weeks, no signs of rejection were detected. (one Subsequent reports It is noted from SANA Biotechnology that the implanted cells are still escaping the patient’s immune system after six months.
The tests were conducted as part of the study and documented the function of the cells: the implanted cells respond to insulin secreted at glucose levels, representing a critical step in controlling diabetes without the need for insulin injection. Four adverse events were recorded during follow-up with the patient, but none were severely or directly associated with modified cells.
The researchers’ ultimate goal is to apply gene editing of immune diaphragms to stem cells, which are able to reproduce and distinguish themselves into other cell types in the body, which are then directed into insulin-secreting islet cells. “The advantage of engineered low immune stem cells is that when these stem cells proliferate and create new cells, new cells will also decrease,” Schrepfer explained in A. Cedars-Sinai Q+a Earlier this year.
Traditionally, transplanting foreign cells into patients requires suppressing the patient’s immune system to avoid being rejected. This has great risks: infection, toxicity and long-term complications. “It was frustrating for me to see patients die of rejection or severe complications due to immunosuppression,” Schrepfer told Cedars-Sinai. “I decided to focus my career on developing strategies to overcome the inhibitory effects of immunosuppressive drugs.”
Although the study marks a milestone in finding treatments for type 1 diabetes, it is important to note that this study involved a participant who received low doses of cells in a short period of time – not enough that patients no longer need to control their blood sugar with injected insulin. Magazine Editorial nature It also said that some independent research groups failed to work to confirm that SANA’s approach provides edited cells with the ability to evade the immune system.
SANA will seek to conduct more clinical trials starting next year. Without neglecting the criticism and limitations of the current study, the possibility that transplanted cells are modified to be invisible to the immune system opens up a very promising horizon in regenerative medicine.
This story originally appeared in wired español and has been translated into Spanish.
