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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
"vector-mediated insertional mutagenesis has been reported in some individuals"
symptom
severe combined immunodeficiency
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