Beta cell programming for treatment of diabetes

Objective

Diabetes is a frequent chronic disease that reduces quality of life and increases risks for life-threatening complications. Its onset in younger patients is caused by massive losses in insulin-producing beta cells. Regenerating a functional beta cell mass is thus a major goal in biomedicine and in society. Beta cell grafts prepared from human pancreases can cure the disease but development of this form of beta cell therapy is hindered by shortage in donor organs. Our consortium with leading teams in molecul ar, developmental and functional biology has worked out an integrated program to generate insulin-producing beta cells in therapeutic quantities and established interactions to translate knowledge to associated bioindustry and multicenter clinical trials, as well as to society. Nature's biologic program to develop and preserve a functional beta cell mass throughout life is taken as platform for directing strategies towards laboratory production of a therapeutic beta cell mass. Beta cells will be derived fro m embryonic stem cells and from transdifferentiating liver, intestinal and pancreatic exocrine cells. Functional genomics will be used to compare phenotypes of beta cells from new sources with those isolated from the pancreas. This analysis will direct fur ther research and judge on the start of preclinical testing. It will also generate new tools and quality control criteria that will allow standardization of ongoing trials and adjustments in graft biology to increase its long-term survival and function in patients. The consortium considers its plan realistic in its perspective to help develop a cure for diabetes by (re)programming cells for beta cell therapy.

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VRIJE UNIVERSITEIT BRUSSEL

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Pleinlaan 2
Brussels
Belgium

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