期刊
JOURNAL OF CLINICAL INVESTIGATION
卷 126, 期 10, 页码 3651-3660出版社
AMER SOC CLINICAL INVESTIGATION INC
DOI: 10.1172/JCI87439
关键词
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资金
- NIH
- Juvenile Diabetes Research Foundation
- Helmsley Charitable Trust
- California Institute of Regenerative Medicine
- NIH/National Institute of General Medical Sciences UCSD Genetics Training Program [T32 GM008666]
- Glenn/American Federation for Aging Research Scholarship for Research in the Biology of Aging
In the past decade, new approaches have been explored that are aimed at restoring functional beta cell mass as a treatment strategy for diabetes. The two most intensely pursued strategies are beta cell replacement through conversion of other cell types and beta cell regeneration by enhancement of beta cell replication. The approach closest to clinical implementation is the replacement of beta cells with human pluripotent stem cell-derived (hPSC-derived) cells, which are currently under investigation in a clinical trial to assess their safety in humans. In addition, there has been success in reprogramming developmentally related cell types into beta cells. Reprogramming approaches could find therapeutic applications by inducing beta cell conversion in vivo or by reprogramming cells ex vivo followed by implantation. Finally, recent studies have revealed novel pharmacologic targets for stimulating beta cell replication. Manipulating these targets or the pathways they regulate could be a strategy for promoting the expansion of residual beta cells in diabetic patients. Here, we provide an overview of progress made toward beta cell replacement and regeneration and discuss promises and challenges for clinical implementation of these strategies.
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