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Enhanced brain distribution of modified aspartoacylase.
[canavan disease]
Canavan
disease
is
a
fatal
neurological
disorder
caused
by
defects
in
the
gene
that
produces
the
enzyme
aspartoacylase
.
Enzyme
replacement
therapy
can
potentially
be
used
to
overcome
these
defects
if
a
stable
enzyme
form
that
can
gain
access
to
the
appropriate
neural
cells
can
be
produced
.
Achieving
the
proper
cellular
targeting
requires
a
modified
form
of
aspartoacylase
that
can
traverse
the
blood
-
brain
barrier
.
A
PEGylated
form
of
aspartoacylase
that
shows
dramatic
enhancement
in
brain
tissue
access
and
distribution
has
been
produced
.
While
the
mechanism
of
transport
has
not
yet
been
established
,
this
modified
enzyme
is
significantly
less
immunogenic
than
unmodified
aspartoacylase
.
These
improved
properties
set
the
stage
for
more
extensive
enzyme
replacement
trials
as
a
possible
treatment
strategy
.
Diseases
Validation
Diseases presenting
"distribution has been produced"
symptom
canavan disease
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