期刊
GENE THERAPY
卷 12, 期 2, 页码 101-107出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302412
关键词
diabetes; cell therapy; beta-cells; islet; stem cells
类别
资金
- NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES [R01DK068037] Funding Source: NIH RePORTER
- NIDDK NIH HHS [DK068037] Funding Source: Medline
Diabetes mellitus has long been targeted, as yet unsuccessfully, as being curable with gene therapy. The main hurdles have not only been vector-related toxicity but also the lack of physiological regulation of the expressed insulin. Recent advances in understanding the developmental biology of beta-cells and the transcriptional cascade that drives it have enabled both in vivo and ex vivo gene therapy combined with cell therapy to be used in animal models of diabetes with success. The associated developments in the stem cell biology and immunology have opened up further opportunities for gene therapy to be applied to target autoimmune diabetes.
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