CRISPR provides a new hope for the treatment of diabetes

CRISPR gene editing technology It has shown its revolutionary potential in recent years: it has been used to treat rare diseases, adaptation of crops to withstand excessive climate change or even spider web discoloration. But the most hope is that this technology will help to find a cure for a global disease such as diabetes. A new study refers to that direction.

For the first time, the researchers have been able to plant pancreatic cells edited with CRISPR in 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 is unable to regulate blood sugar. If no measures to manage glucose levels in other ways (typically, insulin injection), this can lead to damage to the nerves and organs – especially the heart, kidneys and eyes. Approximately 9.5 million people worldwide have type 1 diabetes.

In this experiment, edited cells produced months after insulin implantation, without the need for receptor to prevent the body from attacking the cells. CRISPR technology allows researchers to dedicate genetic modified cells to camouflage to escape the diagnosis.

This study, published last month in the Journal of New England MedicineWith Show the details of the step -by -step method. First, the pancreatic island cells were taken from a deceased donor without diabetes and then changed by the CRISPR-CAS12B gene editing method to allow them to escape the patient’s immune response. Changed cells such as “hypoimmune” explain Sonja Schrepper, professor of the Cedars-Sinai Medical Center in California and the founder of Sana’s biotechnology, a company that has developed this treatment.

The edited cells were then planted to the patient’s forearm and after 12 weeks, no signs of rejection were observed. (The next report on Sana’s biotechnology points out that the implanted cells were still fleeing the patient’s immune system after six months.)

Experiments are recorded as part of the study that cells are functional: insulin implanted cells in response to glucose levels, and show a key step in controlling diabetes without the need for insulin injection. Four side effects were recorded during follow -up with the patient, but none of them were seriously or directly related to modified cells.

The ultimate goal of the researchers is to use the body’s immune gene editing in stem cells that are capable of reproducing and distinguishing themselves into other cells in the body-and then directing their progress to the islands of insulin secretion. “The advantage of the engineering hypoimmune stem cells is that when these stem cells reproduce and create new cells, new cells are also exposed to blood pressure,” the Shepherd explained early this year.

Traditionally, external cell transplantation to the patient needs to suppress the immune system to prevent them from rejecting. This has significant risks: infection, toxicity, and long -term complications. “Seeing patients from the rejection or severe complications caused by the suppression of the immune system was disappointing for me, and I decided to focus my career in formulating strategies to overcome immune-free immune drugs,” Shapes told Cedars-Sinai.

Although this study is a turning point in search of type 1 diabetes, it should be noted that this study contains a participant, who has received a low -cell dose for a short time – sufficiently not need to control his blood sugar with insulin. A editorial by the journal Nature also says that some independent research groups have failed to confirm that the SANA method offers edited cells with the ability to escape the immune system.

Sana will seek more clinical tests than next year. Without the criticism and limitations of the present study, the possibility of transplanting cells for invisible immune system for the immune system will open a very promising horizon in the regenerative medicine.

This story appeared first Wired en español And translated from Spanish.