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A random Abstract
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Somatic gene therapy for phenylketonuria and other hepatic deficiencies.
[classical phenylketonuria]
Gene
therapy
is
the
delivery
of
genetic
material
to
specific
cell
types
of
an
organism
to
alter
its
physiology
or
function
.
This
technology
is
being
explored
as
a
means
of
treating
diseases
caused
by
deficiencies
of
hepatic
gene
products
.
The
two
diseases
being
used
as
models
for
hepatic
gene
therapy
are
classical
phenylketonuria
(
PKU
)
and
haemophilia
B
.
Vectors
derived
from
adenoviruses
can
be
used
to
completely
correct
these
diseases
in
animal
models
.
The
phenotypic
correction
generated
in
these
studies
is
transient
,
and
can
not
be
duplicated
by
vector
readministration
.
The
transient
nature
of
transgene
expression
results
from
the
destruction
of
the
virally-transduced
cells
by
a
cellular
immune
response
directed
against
the
late
viral
gene
products
that
are
also
expressed
in
the
target
cells
.
The
inability
to
repeatedly
administer
virus
is
caused
by
a
humoral
immune
response
directed
against
viral
proteins
present
at
the
time
of
infusion
.
If
the
host
immune
response
is
suppressed
,
transgene
expression
can
persist
for
6
months
or
more
.
These
findings
suggest
that
host
immunomodulation
in
combination
with
further
modification
of
the
adenoviral
vector
to
reduce
or
eliminate
late
viral
gene
expression
may
permit
long
-term
expression
of
potentially
therapeutic
gene
products
in
mammalian
liver
.
