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A random Abstract
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Clinical applications involving CNS gene transfer.
[canavan disease]
Diseases
of
the
central
nervous
system
(
CNS
)
have
traditionally
been
the
most
difficult
to
treat
by
traditional
pharmacological
methods
,
due
mostly
to
the
blood
-
brain
barrier
and
the
difficulties
associated
with
repeated
drug
administration
targeting
the
CNS
.
Viral
vector
gene
transfer
represents
a
way
to
permanently
provide
a
therapeutic
protein
within
the
nervous
system
after
a
single
administration
,
whether
this
be
a
gene
replacement
strategy
for
an
inherited
disorder
or
a
disease-modifying
protein
for
a
disease
such
as
Parkinson
's
.
Gene
therapy
approaches
for
CNS
disorders
has
evolved
considerably
over
the
last
two
decades
.
Although
a
breakthrough
treatment
has
remained
elusive
,
current
strategies
are
now
considerably
safer
and
potentially
much
more
effective
.
This
chapter
will
explore
the
past
,
current
,
and
future
status
of
CNS
gene
therapy
,
focusing
on
clinical
trials
utilizing
adeno-associated
virus
and
lentiviral
vectors
.