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Intracerebral cell transplantation therapy for murine GM1 gangliosidosis.
[gm1 gangliosidosis]
We
performed
a
cell
transplantation
study
to
treat
the
brain
involvement
in
lysosomal
storage
diseases
.
We
used
acid
beta
-galactosidase
knock-out
mice
(
BKO
)
from
C
5
7
BL
/
6
as
recipients
.
To
minimize
immune
responses
,
we
used
cells
derived
from
transgenic
mice
of
C
5
7
BL
/
6
overexpressing
the
normal
human
beta
-galactosidase
.
Fetal
brain
cells
(
FBC
)
,
bone
marrow-derived
mesenchymal
stem
cells
(
MSC
)
,
and
mixed
FBC
and
MSC
cells
were
prepared
and
injected
into
the
ventricle
of
newborn
BKO
mouse
brain
.
The
mice
were
examined
at
1
,
2
,
4
,
and
8
weeks
and
6
months
after
injection
.
In
each
experiment
,
the
injected
cells
migrated
into
the
whole
brain
effectively
and
survived
for
at
least
8
weeks
.
Decrease
in
ganglioside
GM
1
level
was
also
observed
.
FBC
could
survive
for
6
months
in
recipient
brain
.
However
,
the
number
of
transplanted
FBC
decreased
.
In
the
brains
of
MSC
-
or
mixed
cell-treated
mice
,
no
grafted
cells
could
be
found
at
6
months
.
To
achieve
sufficient
long
-term
effects
on
the
brain
,
a
method
of
steering
the
immune
response
away
from
cytotoxic
responses
or
of
inducing
tolerance
to
the
products
of
therapeutic
genes
must
be
developed
.
Diseases
Validation
Diseases presenting
"mixed cell-treated mice"
symptom
gm1 gangliosidosis
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