From Maine, USA:
My granddaughter (2 years old) was within the past week told she has Type 1 diabetes. I have heard about some new research involving gene therapy. Can you tell me more?
When you consider a possible gene therapy for Type 1 diabetes you must first remember that diabetes is not a single gene disease but rather a combination of some genetics and bad luck (environmental factors) that together conspire to cause diabetes.
Genetic susceptibility is obviously conferred by our parents and, although we now know quite a lot about the various genes involved, we still have more to find out (more than 20 genes are currently known to have an influence upon diabetes risk), especially on how genes influence the autoimmune process that is believed to lead to diabetes. In fact, we know that both whole pancreas and islets transplantation, and even regenerated islets from pancreatic duct tissue, need exogenous immune therapy to avoid the host immune system to destroy the "new" beta cells.
Gene therapy for Type 1 diabetes is nowadays directed towards the genetic engineering of beta cells from animal sources to make them able to recognize glucose and to release adequate amount of insulin as the human cells do. In order to block the immune system from attacking the cells, they have been encapsulated. So far, this has been obtained only with rat cells.
To avoid the need of any immune protection, a different approach that is being considered is to manipulate cells that normally don't produce insulin (e.g., cells from the skin, liver and muscle) taken from the same patient and make them able to produce and release insulin from proinsulin. This has been achieved in animal models with fibroblasts although at the price of hypoglycemia because these cells miss the glucose sensor that tune precisely the insulin production and its release from normal beta cells. Studies are currently under progress to transfer to these cells the genetic information for the glucose sensor. You will be pleased to hear a great deal of effort in this field in the coming years.
Original posting 19 Mar 1998
Posted to Research: Cure
Last Updated: Tuesday April 06, 2010 15:08:56
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