A man from type 1 diabetes mellitus became the world’s first patient to start producing his own insulin after transplantation of genetically modified cells.
Type 1 diabetes occurs, when the immune system destroys specialized cells in the pancreas, called islet cells. These cells are responsible for the production of insulin, a hormone that regulates blood sugar level.
Usually, treatment consists of constant injections of synthetic insulin. However, islet cell transplantation can provide longer supply of insulin for patients with type 1 diabetes.
After transplantation, the patient’s immune system may perceive the transplanted cells as foreign and attack these tissues. As a result, transplant patients are forced to take immunosuppressants for life, making them vulnerable to infections.
To solve this problem, Swedish and American scientists transplanted islet cells that had been genetically modified using a gene editor CRISPR, in order to suppress rejection by the immune system. 12 weeks after transplantation, the patient’s body continues to produce insulin and does not meet a response from the immune system.
The authors of the study note, that although it is preliminary, it still demonstrates that genetic engineering of cell transplantation can solve the problems of immune response. The researchers used CRISPR to create three changes in the genetic code of donor cells to reduce the likelihood of rejection by the immune system of the future host.
Two of the changes involved a decrease in the level of proteins on the cell surface that signal to white blood cells whether a cell is foreign. Another change increased the production of the CD47 protein, which counteracts attack from the immune system.
The genetically edited cells were then injected into the man’s forearm. His body did not react to the modified cells, and the surviving cells began to produce insulin as usual.
The man received a small dose of the edited cells and still needs a daily dose of insulin. The next step for the researchers will be to conduct additional studies to find out if the cells are able to survive in the long term, which will facilitate the treatment of the disease and potentially provide a cure. They also need to conduct additional trials to determine whether this approach is effective in other patients.
The results of the study were published in the journal New England Journal of Medicine
Source: LiveScience
Spelling error report
The following text will be sent to our editors: