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Effect of intrastriatal mesenchymal stromal cell injection on progression of a murine model of Krabbe disease.
[krabbe disease]
One
of
a
family
of
devastating
lysosomal
storage
disorders
,
Krabbe
disease
is
characterized
by
demyelination
,
psychosine
accumulation
,
and
inflammation
.
Affected
infants
rarely
survive
longer
than
2
years
.
Using
the
twitcher
mouse
model
of
the
disease
,
this
study
evaluated
the
potential
of
intrastriatal
injection
of
adipose
or
bone
marrow-derived
mesenchymal
stromal
cells
(
MSCs
)
as
a
treatment
option
.
Neonatal
pups
were
injected
with
MSCs
at
3
-
4
days
of
age
and
subjected
to
a
battery
of
behavioral
tests
beginning
at
15
days
.
While
MSC
injection
failed
to
increase
lifespan
of
twitchers
,
improvements
in
rotarod
performance
and
twitching
severity
were
observed
at
27
-
38
days
of
age
using
MSCs
derived
from
bone
marrow
.
This
study
tested
several
different
tasks
developed
in
adult
mice
for
evaluation
of
disease
progression
in
immature
twitchers
.
Rotarod
was
both
reliable
and
extremely
sensitive
.
Automated
gait
analysis
using
the
Treadscan
program
was
also
useful
for
early
evaluation
of
differences
prior
to
overt
gait
dysfunction
.
Finally
,
this
study
represents
the
first
use
of
the
Stone
T
-
maze
in
immature
mice
.
Validation
of
rotarod
and
automated
gait
analysis
for
detection
of
subtle
differences
in
disease
progression
is
important
for
early
stage
efforts
to
develop
treatments
for
juvenile
disorders
.
