Gerosper Genes’s Liberation Technology It has shown its revolutionary capabilities in recent years: it has been used Treat rare diseasesTo Copy air conditioning To bear the maximum climate change, or even discoloration From the spider network. But the greatest hope is that This technology It will help find a treatment for a global disease, such as Diabetic. A new study in this direction.
For the first time, the researchers succeeded in implanting the pancreas cells that CRISBR release in a man with type 1 diabetes, an autoimmune disease where the immune system attacks insulin production cells in the pancreas. Without insulin, the body is unable to regulate blood sugar. If steps are not taken to manage glucose levels by other means (usually, by injecting insulin), this can lead to nerve damage and organs – especially the heart, kidneys and eyes. About 9.5 million people around the world suffer from type 1 diabetes.
In this experiment, the cells that were released by insulin produced for several months after planting them, without the need for the recipient to take any immunosuppressive medications to prevent their body from attacking cells. CRISPR technology allowed researchers to unify genetically modified cells with camouflage to evade detection.
The study published last month in New England Magazine for Medicineand Step -step procedure details. First, the cells of the pancreatic island were taken from a deceased donor without diabetes, then it was changed with the CRISPR-CAS12B genetics technology to allow them to evade the immune response to a diabetic patient. The changes that have been changed are said to be “immunodeficiency”.
Then the modified cells were planted in the patient’s forearm muscle, and after 12 weeks, no signs of rejection were discovered. (A. The subsequent report From Sana Biotechnology notes that the cultivated cells are still evading the patient’s immune system after six months.)
The tests were recorded as part of the study that the cells were functional: the transplanted cells secrete insulin in response to glucose levels, which represents a major step towards controlling diabetes without the need for insulin injection. Four negative events were recorded during the follow -up with the patient, but none of them were dangerous or directly linked to the modified cells.
The ultimate goal of researchers is to apply immune genes adjustments to stem cells-which have the ability to reproduce and distinguish themselves in other cells within the body-and to direct their development into insulin island cells. “The feature of immune stem cell engineering is that when these stem cells multiply and the creation of new cells, new cells are also immunodeficiency,” explained Sherbar in A. Cedars-Sinai Q+A Earlier this year.
Traditionally, the implantation of foreign cells is required to the patient’s suppression of the patient’s immune system to avoid rejecting them. This carries great risks: infections, toxicity and long -term complications. “The vision of patients dies from rejection or severe complications from immunity suppression was frustrating for me, and I decided to focus on my career on strategies for developing immunosuppressive rejection without immunosuppressive drugs,” said Shrebar Siders-Cineis.
Although the research represents a milestone in the search for treatments of type 1 diabetes, it is important to note that the study included one of the participants, which received a low dose of cells for a short period – it is not enough for the patient to need to control blood sugar with the syringe insulin. An editorial from a magazine nature It also says that some independent research groups have failed in their efforts to confirm that the Sana method provides modified cells the ability to evade the immune system.
Sana will look at more clinical experiences that start next year. Without overcoming the current criticism and restrictions of the study, the possibility of planting modified cells to be invisible to the immune system opens a very promising horizon in renewal medicine.
This story was originally appeared on Wireless En español It was translated from Spanish.
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