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The genetically modified polysialylated form of neural cell adhesion molecule-positive cells for potential treatment of X-linked adrenoleukodystrophy.
[x-linked adrenoleukodystrophy]
Cell
transplantation
of
myelin-producing
exogenous
cells
is
being
extensively
explored
as
a
means
of
remyelinating
axons
in
X-
linked
adrenoleukodystrophy
.
We
determined
whether
3
,
3
'
,
5
-
Triiodo-
L-
thyronine
(
T
3
)
overexpresses
the
ABCD
2
gene
in
the
polysialylated
(
PSA
)
form
of
neural
cell
adhesion
molecule
(
NCAM
)
-
positive
cells
and
promotes
cell
proliferation
and
favors
oligodendrocyte
lineage
differentiation
.
P
SA-NCAM
+
cells
from
newborn
Sprague-
Dawley
rats
were
grown
for
five
days
on
uncoated
dishes
in
defined
medium
with
or
without
supplementation
of
basic
fibroblast
growth
factor
(
bFGF
)
and
/
or
T
3
.
Then
,
PSA-NCAM
+
spheres
were
prepared
in
single
cells
and
transferred
to
polyornithine
/
fibronectin-coated
glass
coverslips
for
five
days
to
determine
the
fate
of
the
cells
according
to
the
supplementation
of
these
molecules
.
T
3
responsiveness
of
ABCD
2
was
analyzed
using
real-time
quantitative
polymerase
chain
reaction
,
the
growth
and
fate
of
cells
were
determined
using
5
-
bromo-
2
-
deoxyuridine
incorporation
and
immunocytochemistry
,
respectively
.
Results
demonstrated
that
T
3
induces
overexpression
of
the
ABCD
2
gene
in
PSA-NCAM
+
cells
,
and
can
enhance
PSA-NCAM
+
cell
growth
in
the
presence
of
bFGF
,
favoring
an
oligodendrocyte
fate
.
These
results
may
provide
new
insights
into
investigation
of
PSA-NCAM
+
cells
for
therapeutic
application
to
X-
linked
adrenoleukodystrophy
.
Diseases
Validation
Diseases presenting
"growth factor"
symptom
22q11.2 deletion syndrome
achondroplasia
adrenal incidentaloma
aniridia
cadasil
cholangiocarcinoma
coats disease
dedifferentiated liposarcoma
dentin dysplasia
dentinogenesis imperfecta
dystrophic epidermolysis bullosa
esophageal carcinoma
esophageal squamous cell carcinoma
hodgkin lymphoma, classical
holt-oram syndrome
inclusion body myositis
kallmann syndrome
krabbe disease
liposarcoma
lymphangioleiomyomatosis
oculocutaneous albinism
oral submucous fibrosis
pleomorphic liposarcoma
primary effusion lymphoma
primary hyperoxaluria type 1
severe combined immunodeficiency
systemic capillary leak syndrome
von hippel-lindau disease
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
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