Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Engraftment of human iPS cells and allogeneic porcine cells into pigs with inactivated RAG2 and accompanying severe combined immunodeficiency.
[severe combined immunodeficiency]
Pigs
with
severe
combined
immunodeficiency
(
SCID
)
may
provide
useful
models
for
regenerative
medicine
,
xenotransplantation
,
and
tumor
development
and
will
aid
in
developing
therapies
for
human
SCID
patients
.
Using
a
reporter-guided
transcription
activator-like
effector
nuclease
(
TALEN
)
system
,
we
generated
targeted
modifications
of
recombination
activating
gene
(
RAG
)
2
in
somatic
cells
at
high
efficiency
,
including
some
that
affected
both
alleles
.
Somatic-cell
nuclear
transfer
performed
with
the
mutated
cells
produced
pigs
with
RAG
2
mutations
without
integrated
exogenous
DNA
.
Biallelically
modified
pigs
either
lacked
a
thymus
or
had
one
that
was
underdeveloped
.
Their
splenic
white
pulp
lacked
B
and
T
cells
.
Under
a
conventional
housing
environment
,
the
biallelic
RAG
2
mutants
manifested
a
"
failure
to
thrive
"
phenotype
,
with
signs
of
inflammation
and
apoptosis
in
the
spleen
compared
with
age-matched
wild-
type
animals
by
the
time
they
were
4
wk
of
age
.
Pigs
raised
in
a
clean
environment
were
healthier
and
,
following
injection
of
human
induced
pluripotent
stem
cells
(
iPSCs
)
,
quickly
developed
mature
teratomas
representing
all
three
germ
layers
.
The
pigs
also
tolerated
grafts
of
allogeneic
porcine
trophoblast
stem
cells
.
These
SCID
pigs
should
have
a
variety
of
uses
in
transplantation
biology
.
Diseases
Validation
Diseases presenting
"will aid in developing therapies for human scid patients"
symptom
severe combined immunodeficiency
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom