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A random Abstract
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Novel mutations in RAG1/2 and ADA genes in Israeli patients presenting with T-B-SCID or Omenn syndrome.
[omenn syndrome]
The
relative
frequency
of
the
different
forms
of
SCID
may
vary
in
different
countries
.
The
most
frequent
form
in
Israel
is
the
autosomal-recessive
T
-B-
SCID
or
Omenn
syndrome
while
X-
linked
SCID
is
rare
.
We
report
our
immunological
and
genetic
analyses
in
multicentre
study
of
patients
presenting
with
either
T
-B-
SCID
or
Omenn
syndrome
.
Among
16
patients
,
we
identified
7
novel
mutations
in
6
patients
.
In
the
RAG
1
gene
we
detected
two
novel
mutations
:
L
454
Q
and
469
fs-
4
bpdel
.
In
the
RAG
2
gene
:
3
novel
mutations
:
D
65
Y
,
G
157
V
,
and
E
480
X
.
One
T
-B-
SCID
patient
was
found
to
be
a
compound
heterozygote
for
new
mutations
in
the
ADA
gene
:
W
264
X
and
R
235
W
.
Prenatal
diagnosis
was
performed
in
8
families
while
others
refused
due
to
religious
reasons
.
Identification
of
the
new
mutations
expands
our
knowledge
regarding
the
unique
features
of
SCID
phenotype
in
Israel
and
may
help
the
families
seeking
for
genetic
counseling
.
Diseases
Validation
Diseases presenting
"prenatal diagnosis"
symptom
22q11.2 deletion syndrome
achondroplasia
adrenomyeloneuropathy
alexander disease
alpha-thalassemia
aromatase deficiency
benign recurrent intrahepatic cholestasis
cadasil
canavan disease
classical phenylketonuria
cohen syndrome
congenital adrenal hyperplasia
congenital diaphragmatic hernia
congenital toxoplasmosis
cystinuria
dentinogenesis imperfecta
epidermolysis bullosa simplex
harlequin ichthyosis
holt-oram syndrome
homocystinuria without methylmalonic aciduria
hydrocephalus with stenosis of the aqueduct of sylvius
junctional epidermolysis bullosa
kindler syndrome
krabbe disease
lamellar ichthyosis
megacystis-microcolon-intestinal hypoperistalsis syndrome
monosomy 21
neonatal adrenoleukodystrophy
oculocutaneous albinism
omenn syndrome
phenylketonuria
primary hyperoxaluria type 1
pyruvate dehydrogenase deficiency
severe combined immunodeficiency
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
zellweger syndrome
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