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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
"including some that affected both alleles"
symptom
severe combined immunodeficiency
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