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 Investigators:
Terrence Flotte, Nicholas
Muzyczka
Many genes have been discovered that might be helpful
in preventing diabetes or its complications. Some of these genes
could be particularly helpful in patients who receive either kidney
transplants or transplants of insulin-producing, or "islet" cells.
In order to function, however, these genes must be inserted into
the islet cells or the kidney cells. This is accomplished with carriers,
called "vectors".
Our group has developed one such vector, known as
"AAV". AAV can permanently insert genes into cells without causing
side effects, but the ability of AAV to enter certain cells has
been limited. In islets, for instance, about 10,000 AAV particles
are needed to insert a single gene. We believe that this could be
due to a lack of a certain docking protein or "receptor" on the
surface of the islet cells. We have found a way to change the coat
of the AAV so that it can use other receptors to enter cells.
In the course of these studies we will attempt to
improve the efficiency of AAV-based gene delivery in islet cells
and kidney cells through these kinds of modifications. We will also
work to optimally control the switches that turn the newly inserted
genes on or off. This could be very important since some of these
genes control the immune system. We will plan to share all of our
newly improved AAV vector components with all of the other scientists
in the program and so improve their chances of successfully accomplishing
gene therapy.
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