Embryonic Stem Cells and Disease
Brought to you by the Howard Hughes Medical
Institute, this lecture by Dr. Douglas A. Melton is part of a series
of lectures devoted to a discussion about the nature of embryonic
stem cells and their potential use in the treatment of human
disease. Understanding the mechanisms by which particular cell types
are generated are of primary concern to be able to fully harness the
medicinal potential of embryonic stem cells.
HHMI description:
There are two main approaches to using stem cells to fight human
diseases: develop stem cells to produce therapeutic replacement
cells and study stem cells as a model for understanding the biology
of a disease. Significant progress has been made in producing stem
cell lines that, for example, participate in the regeneration of
damaged nervous tissue. Many human diseases, such as juvenile
diabetes (type 1 diabetes), involve malfunctioning genes and
environmental triggers. Usually, a specific type of cell is
primarily affected by the disease, and the cellular dysfunction
produces the symptoms. In juvenile diabetes, the insulin-producing
islet cells of the pancreas are destroyed. Insulin is critical to
the proper regulation of sugar by the body, and its absence causes
the severe condition called diabetes. Researchers want to coax
embryonic stem cells into becoming healthy insulin-producing cells.
These cells might then be transplanted into people with diabetes to
produce the insulin they lack. Researchers are also interested in
producing stem cells that malfunction exactly like the diseased
cells in order to understand fundamental aspects of the disease and
also to test treatments.