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Regulation of the adrenoleukodystrophy-related gene (ABCD2): focus on oxysterols and LXR antagonists.
[x-linked adrenoleukodystrophy]
The
regulation
of
the
ABCD
2
gene
is
recognized
as
a
possible
therapeutic
target
for
X-
linked
adrenoleukodystrophy
,
a
rare
neurodegenerative
disease
caused
by
mutations
in
the
ABCD
1
gene
.
Up-regulation
of
ABCD
2
expression
has
indeed
been
demonstrated
to
compensate
for
ABCD
1
deficiency
,
restoring
peroxisomal
β-oxidation
of
very
-
long
-chain
fatty
acids
.
Besides
the
known
inducers
of
the
ABCD
2
gene
(
phenylbutyrate
and
histone
deacetylase
inhibitors
,
fibrates
,
dehydroepiandrosterone
,
thyroid
hormone
and
thyromimetics
)
,
this
review
will
focus
on
LXR
antagonists
and
22
S-
hydroxycholesterol
,
recently
described
as
inducers
of
ABCD
2
expression
.
Several
LXR
antagonists
have
been
identified
and
their
possible
indication
for
neurodegenerative
disorders
will
be
discussed
.