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My name is Roland Herzog.
I'm a professor at the University
of Florida Department of Pediatrics
at the Powell Gene Therapy Center
where we are developing gene
therapies for genetic
disease using viral vectors
to transfer our therapeutic genes.
One of the key questions that
we're addressing in my laboratory
is what do we have to do to
avoid rejection of our therapy
by the immune system?
So understanding the adaptive immune
response to viral gene transfer
is key for sustained therapy and
successful delivery of our genes.
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The concept of viral gene
transfer is quite simple.
We use recombinant viruses
which we engineered to contain
a therapeutic gene that could
correct a genetic defect.
Viruses have evolved to effectively
infect a variety of cell types.
So we can choose and
design these viral vectors
to infect a certain type
of cells and express
our therapeutic gene in those cells.
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However, a potential
complication of this approach
is that the immune system has
evolved to fend off versus
and to recognize viruses
as invading pathogens.
Shown here is an example of
a muscle fiber, shown in red,
which expresses a gene
that we transferred
in using a viral vector.
And this cell is being surrounded
by these green cells, which
are cytotoxic T lymphocytes--
CD8 positive T cells that
attack and destroy
this muscle fiber.
So in this case, the immune
system has rejected our therapy.