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Trends Endocrinol Metab. 2006 Apr;17(3):92-100. Epub 2006 Feb 28.

Gene therapy for diabetes: reinventing the islet.

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Departments of Medicine and Molecular and Cellular Biology, Baylor College of Medicine, One Baylor Plaza, Houston, TX 77030, USA.


A cure for type 1 (insulin dependent) diabetes might be found in generating surrogate insulin-producing cells to replace beta cells. A gene therapy strategy using constructs designed to allow glucose-regulated insulin transcription when delivered to non-pancreatic tissues has not fully recreated the stringent control of blood glucose provided by the beta cell. A more promising gene therapy approach has been to express pancreatic endocrine developmental factors, such as PDX-1, NeuroD/BETA2 and Neurogenin 3, to promote differentiation of non-endocrine cells towards a beta cell or islet phenotype, enabling these cells to synthesize and secrete insulin in a glucose-regulated manner. Further research is necessary, however, to better define the most effective pro-endocrine factors and the most amenable cell types to achieve transdifferentiation for beta cell replacement.

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