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Induced pluripotent stem cells for modeling of pediatric neurological disorders.
[x-linked adrenoleukodystrophy]
The
pathophysiological
mechanisms
underlying
childhood
neurological
disorders
have
remained
obscure
due
to
a
lack
of
suitable
disease
models
reflecting
human
pathogenesis
.
Using
induced
pluripotent
stem
cell
(
iPSC
)
technology
,
various
neurological
disorders
can
now
be
extensively
modeled
.
Specifically
,
iPSC
technology
has
aided
the
study
and
treatment
of
early
-onset
pediatric
neurodegenerative
diseases
such
as
Rett
syndrome
,
Down
syndrome
,
Angelman
syndrome
.
Prader-
Willi
syndrome
,
Friedreich
's
ataxia
,
spinal
muscular
atrophy
(
SMA
)
,
fragile
X
syndrome
,
X-
linked
adrenoleukodystrophy
(
ALD
)
,
and
SCN
1
A
gene
-related
epilepsies
.
In
this
paper
,
we
provide
an
overview
of
various
gene
delivery
systems
for
generating
iPSCs
,
the
current
state
of
modeling
early
-onset
neurological
disorders
and
the
ultimate
application
of
these
in
vitro
models
in
cell
therapy
through
the
correction
of
disease-
specific
mutations
.
Diseases
Validation
Diseases presenting
"the ultimate application of these in vitro models in cell therapy through the correction of disease-specific mutations"
symptom
x-linked adrenoleukodystrophy
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