| Investigators: Richard Synder
The purpose of Core B is to produce the carriers or
"vectors" that insert good genes into cells to prevent or reverse
the complications that often occur when people with diabetes receive
transplants. Scientists in other portions of the program have good
evidence about which genes might help the transplanted insulin-producing
cells, called "islet" cells, and/or transplanted kidneys to live
longer and function better in diabetes patients.
In order for these good genes to function, they must
be inserted into the right cells, however. That can best be accomplished
with one specific vector, a harmless modified virus called "AAV".
Core B has had many years of experience with making AAV vectors
for scientists in Florida, other parts of the US, and throughout
the world. We have developed better production methods and better
ways to purify the AAV vectors away from another contaminating substances
that might cause side effects or impair their effectiveness. Using
these methods we can produce batches of AAV vector that are very
potent and very pure, thus allowing for the best chance of success
in all of the other parts of the program.
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