For years, attempts have been made to cure type 1 diabetes, with those affected relying on insulin injections once the disease begins. Now researchers could have taken a big step closer to their goal. For the first time, it is possible to transplant insulin-producing cells from a donor’s pancreas to a diabetic patient without causing a rejection reaction.
Until now, all patients who received donor islet cells had to be treated the same as other organ transplant recipients to prevent rejection: lifelong with strong drugs that significantly weaken the immune system with possible serious long-term complications. Swedish and American researchers have now succeeded in developing so-called hypoimmune insulin-producing cells from the pancreas of a healthy donor.
In December last year, a patient with type 1 diabetes was treated with such genetically modified cells for the first time at Uppsala University Hospital. “Per-Ola Carlsson’s team, which has experience with 60 conventional islet cell transplants since 2001 (with the need for subsequent immunosuppression; note), implanted the ‘pseudo islet cells’ in the upper arm of the unknown patient. The transplant has survived for four weeks so far, even though the patient has not received immunosuppressants,” the clinic said in a press release.
The use of “genetic scissors” to eliminate foreign tissue features
Behind the medical success lies a technology based on the ‘gene scissors’ CRISPR-Cas9. In the current case, tissue features that make the cells appear “foreign” to the recipient’s immune system and trigger an immediate and destructive rejection response were eliminated. This happened in several steps. Using the CRISPR-Cas9 gene scissors, two immunologically active genes (B2M and CIITA) were removed from the genetic material of the donor cells. An extra gene (CD47) was then introduced via gene ferry, the German medical newspaper reported.
In an animal model, the transplantation of such islet cells even crossed the species barrier of two macaques (Rhesus/Java) and cured artificially induced type 1 diabetes for six months, the Sana scientists reported last year in the journal “Cell Stem Cel”.
This is how type 1 diabetes develops
It is not yet known to what extent insulin production has been restarted by the genetically modified islet cells in the treated patient. Details will be published in a specialized publication. The goal would be that people with type 1 diabetes no longer need to inject insulin. A drug for type 1 diabetes, in which an autoimmune reaction destroys insulin-producing cells in the pancreas, would be a huge advance in medicine. The disease usually breaks out in childhood and adolescence, meaning those affected are particularly at risk for long-term complications such as early atherosclerosis, kidney and retina damage later in life. Once diagnosed, they rely on regular insulin injections and constant blood sugar monitoring.
Source: Krone
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