CRISPR gene edition technology has shown his revolutionary potential in recent years: it was used to treat infrequent diseases, to adapt withstand the extremes of climate change, and even to Change color spider network. But the greatest hope is that this technology will support find a cure for a global disease such as diabetes. The novel study indicates in this direction.
For the first time, scientists managed to implant pancreatic cells with the editors of CRISPR in a man with type 1 diabetes, an autoimmune disease in which the immune system attacks the cells producing insulin in the pancreas. Without insulin, the body is not able to regulate blood sugar levels. If they are not taken to manage glucose with other agents (usually by injecting insulin), this can lead to nerve and organs damage – especially hearts, kidneys and eyes. About 9.5 million people around the world have type 1 diabetes.
In this experiment, the edited cells produced insulin for many months after implantation, without the need to take immunosuppressive drugs to stop the body attacking the cells. CRISPR technology has allowed researchers to equip genetically modified cells with camouflage to avoid detection.
Study, published last month The New England Journal of MedicineIN Details of the procedure step by step. First, the cells of the pancreatic islands were taken from the deceased donor without diabetes, and then changed along with the technique of edition of the CRISPR-CAS12B genes to allow them to avoid the immune response of a patient with diabetes. It is said that the cells are changed “Hipoimmune”, explains Sonja Schrepfer, Professor Cedars-Sinai Medical Center in California and the scientific co-founder Sana Biotechnology, a company that developed this treatment.
Edited cells were then implanted in the patient’s forearm muscle, and after 12 weeks no signs of rejection were detected. (AND Another report From Sana Biotechnology notes that the implanted cells still avoided the patient’s immune system after six months.)
Tests conducted as part of the study noted that the cells were functional: implanted insulin cells in response to glucose levels, constituting a key step towards controlling diabetes without the need for insulin injections. Four adverse events were recorded during observation with the patient, but none of them was stern or directly related to modified cells.
The ultimate goal of the scientists is to operate the edition of genes with immune camouflage for stem cells-who have the ability to reproduce and differentiate in other types of cells inside the body-then directing their development to the cells of the islands for insulin secretion. “The advantage of hypoimmunological engineering of stem cells is that when these stem cells proliferate and form new cells, new cells are also hypoimmune,” explained Schrepfe Wa Cedars-Sinai Q+A Early this year.
Traditionally, transplanting foreign cells to the patient required suppressing the patient’s immune system to avoid rejection. This has a significant risk: infections, toxicity and long -term complications. “The vision of patients dying because of rejection or serious complications due to immunosuppression was frustrating for me and I decided to focus my career on developing a strategy to overcome the rejection of immunity without immunosuppressive drugs,” said Schrepfer Cedars-Sinai.
Although the tests mean a milestone in search of treatment of type 1 diabetes, it is crucial to notice that the examination included one participant who received a low dose of cells for a compact time – not enough that the patient no longer had to control blood sugar with injected insulin. Dziennik editors Nature He also says that some independent research groups have not been confirmed that the Sana method provides edited cells with the ability to avoid the immune system.
Sana will want to conduct more clinical trials from next year. Without sustaining criticism and limitations of the current study, the possibility of modifying cells so that they are imperceptible to the immune system, opens a very promising horizon in regenerative medicine.
This story originally appeared Wired in Spanish and was translated from Spanish.