摘要

Purpose of review
Type 1 diabetes is a stereotypical disease amenable to stem cell-based therapy. It is characterized by the loss of a specific cell type and low anatomic complexity of the function in need of restoration. Although embryonic stem cells have the capacity to differentiate into any cell type, inefficient differentiation into beta cells has been common.
Recent findings
An essential challenge is to devise routine methods for generating enriched populations of defined pancreatic precursor or beta cells. Enriched populations of human embryonic stem cell-derived insulin-expressing cells have yet to demonstrate physiologically relevant function. Advances in our understanding of developmental mechanisms are fueling progress towards developing protocols for differentiation of embryonic stem cells into desired lineages for transplantation. Other challenges being intensively studied are the prevention of possible transmission of zoonotic infections and the use of somatic cell nuclear transfer to derive human embryonic stem cells which may be useful for therapeutic applications.
Summary
Many challenges remain in embryonic stem cell research. Initial preclinical and phase I trials should focus on safety with respect to both teratoma formation and genetic stability of cells after transplantation. With in-depth knowledge of the signals and genes involved in endoderm and islet development, it should be possible to recapitulate the process for the production of a virtually unlimited source of transplantable insulin-producing cells.

  • 出版日期2006-2

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