CRISPR gene editing technology Has demonstrated his revolutionary potential in recent years: he has used Ben to three rare dysseas, at Adapt crops At the extremes of climate change, or even Change the color canvas of a spider. But the greatest hope is that this technology will help find a cure for a global disease, such as diabetes. A new study points to this direction.
For the first time, researchers succeed in implanting pancreatic cells edited CRISPR in humans with type 1 diabetes, an autoimmune disease where the immune system attacks insulin producing cells in the pancreas. Without insulin, the body is then unable to regulate blood sugar. If the measures are not taken to manage glucose levels by other means (generally, by injecting insulin), this can cause damage to nerves and organs, in particular the heart, kidneys and eyes. About 9.5 million people worldwide suffer from type 1 diabetes.
In this experience, modified cells have produced insulin for months after being implemented, without the need for the container to take immunosuppressive drugs to prevent their bodies from attacking cells. CRISPR technology has extended researchers to provide genetically modified cells with camouflage to escape detection.
The study, published last month in The New England Journal of Medicine,, Details The procedures step by step. First of all, the cells of the pancreatic islets were taken from a deceased donor without diabetes, then modified with the CRISPR-CAS12B gene editing technique for the Allw to escape the immune response of the diabetes patient. The cells modified as this would be “hypo-immune”, explains Sonja Schrepfer, professor at Cedars-Sinai Medical Center in California and scientific co-founder of Sana Biotechnology, the company that has developed this treatment.
The published cells were then established the patient’s intarm muscle, and after 12 weeks, no sign of rejection was detected. (HAS Subsequent According to Sana Biotechnology notes that implands cells still escape the patient’s immune system after six months.)
The tests carried out as part of the study recruited that the functional wild cells: established cells have secreted insulin in response to glucose levels, representing a key step towards the control of diabetes without the need for intein injections. Four undesirable events were recorded during follow -up with the patient, but none of them was of serus or directly linked to the modified cells.
The ultimate objective of researchers is to apply the modifications of the immuno -camouflage genes to stem cells – which have the capacity to reproduce and differentiate themselves in other types of cells the body – then to direct their development of the cells of condes intin. “The advantage of hypo-immune stem cell engineering is that when these stem cells proliferate and create new cells, new cells are also hypo-immune,” said Schrepfer in a Cedars-sinai q + a Earlier this year.
Traditionally, transplantation of foreign cells into a patient needed to remove the patient’s immune system to prevent them from being rejected. This has significant risks: infections, toxicity and long -term complications. “Seeing the patients die of rejection or serious complications of the immunosuppropression adjustment was frustrating for me, and decided to focus my career on development strategies to go beyond immune rejection with immunosuppressive drugs,” said Schrepfer-Sinai.
Although research marks the milestone in the search for type 1 diabetes treatments, it is important to note that the study involved a participant, who received a low dose of cells for a short period – was not that the patient no longer needed to control his blood sugar with an inject insulin. An editorial by the newspaper Nature Also said that certain independent research groups have failed in their efforts to confirm that the SANA method provides modified cells the skills to escape the immune system.
Sana will seek to conduct more clinical trials from next year. Without neglecting the criticisms and limits of this study, the possibility of transplantation of modified cells to be invisible to the immune system opens a very promising horizon in regenerative medicine.
This story initially looked at Cable EN ESPAñol and was translated from Spanish.