Journal
GENE THERAPY
Volume 22, Issue 1, Pages 1-8Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/gt.2014.93
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Funding
- Australian Postgraduate Award
- Arrow Bone Marrow Transplant Foundation/Hawkesbury Canoe Classic Scholarship
- National Health and Medical Research Council of Australia [352909, 513100]
- Diabetes Australia Research Trust
- Rebecca L Cooper Medical Research Foundation
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Type 1 diabetes results from the autoimmune destruction of the insulin-producing pancreatic beta (beta) cells. Patients with type 1 diabetes control their blood glucose levels using several daily injections of exogenous insulin; however, this does not eliminate the long-term complications of hyperglycaemia. Currently, the only clinically viable treatments for type 1 diabetes are whole pancreas and islet transplantation. As a result, there is an urgent need to develop alternative therapies. Recently, cell and gene therapy have shown promise as a potential cure for type 1 diabetes through the genetic engineering of 'artificial' beta cells to regulate blood glucose levels without adverse side effects and the need for immunosuppression. This review compares putative target cells and the use of pancreatic transcription factors for gene modification, with the ultimate goal of engineering a glucose-responsive 'artificial' beta cell that mimics the function of pancreatic beta cells, while avoiding autoimmune destruction.
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