Rare Diseases Symptoms Automatic Extraction
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Drug delivery to peroxisomes: employing unique trafficking mechanisms to target protein therapeutics.
[zellweger syndrome]
Peroxisomes
are
multifunctional
organelles
of
all
human
cells
,
responsible
for
a
variety
of
essential
biochemical
and
metabolic
processes
including
alpha-
and
beta
-oxidation
of
specific
fatty
acids
,
plasmalogen
biosynthesis
and
glyoxylate
detoxification
.
Inborn
errors
of
biogenesis
or
in
the
ability
to
synthesize
or
properly
traffic
specific
enzymes
to
peroxisomes
result
in
devastating
human
disease
.
The
organelle
has
also
emerged
as
a
contributor
to
cellular
oxidative
stress
through
its
ability
to
generate
hydrogen
peroxide
.
Unlike
most
other
organelles
,
the
peroxisome
's
import
apparatus
will
accommodate
fully
folded
,
oligomeric
and
co
-
factor
-bound
substrates
.
The
strategies
outlined
here
are
designed
to
take
advantage
of
this
unique
mechanism
to
target
protein
therapeutics
.
Emphasis
is
also
placed
on
how
to
deliver
these
bioactive
molecules
into
cells
to
engage
the
peroxisomal
protein
import
machine
.
The
critical
antioxidant
enzyme
catalase
has
been
successfully
delivered
and
targeted
by
many
of
the
approaches
detailed
herein
;
these
examples
will
be
discussed
.