Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus.
[severe combined immunodeficiency]
X-
linked
severe
combined
immunodeficiency
(
SCID
-X
1
)
caused
by
mutations
in
interleukin
2
receptor
gamma
(
IL
2
RG
)
gene
threatens
the
survival
of
affected
boys
during
the
first
year
of
life
unless
hematopoietic
stem
cell
transplantation
is
provided
.
Although
viral
vector-mediated
gene
therapy
has
been
successfully
performed
in
patients
with
no
HLA-matched
donors
,
leukemia
caused
by
vector-mediated
insertional
mutagenesis
has
been
reported
in
some
individuals
.
Transcription
activator-like
effector
nuclease
(
TALEN
)
is
an
artificial
sequence-
specific
endonuclease
that
is
expected
to
revolutionize
the
precise
correction
of
disease-causing
mutations
and
eliminate
the
risk
of
insertional
mutagenesis
.
Here
,
we
report
TALEN-mediated
genome
editing
of
the
IL
2
RG
locus
.
We
transfected
TALENs
along
with
a
targeting
vector
into
Jurkat
cells
,
and
we
confirmed
the
precise
introduction
of
the
exogenous
gene
into
the
IL
2
RG
locus
.
In
addition
,
we
found
that
the
length
of
homology
arm
in
the
targeting
vector
influenced
the
efficiency
of
TALEN-mediated
homologous
recombination
.
Diseases
Validation
Diseases presenting
"affected boys during the first year of life"
symptom
severe combined immunodeficiency
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom