Rare Diseases Symptoms Automatic Extraction
Home
A random Abstract
Our Project
Our Team
Dietary triheptanoin rescues oligodendrocyte loss, dysmyelination and motor function in the nur7 mouse model of Canavan disease.
[canavan disease]
The
inherited
pediatric
leukodystrophy
Canavan
disease
is
characterized
by
dysmyelination
and
severe
spongiform
degeneration
,
and
is
currently
refractory
to
treatment
.
A
definitive
understanding
of
core
disease
mechanisms
is
lacking
,
but
pathology
is
believed
to
result
at
least
in
part
compromised
fatty
acid
synthesis
during
myelination
.
Recent
evidence
generated
in
an
animal
model
suggests
that
the
breakdown
of
N-
acetylaspartate
metabolism
in
CD
results
in
a
heightened
coupling
of
fatty
acid
synthesis
to
oligodendrocyte
oxidative
metabolism
during
the
early
stages
of
myelination
,
thereby
causing
acute
oxidative
stress
.
We
present
here
the
results
of
a
dietary
intervention
designed
to
support
oxidative
integrity
during
developmental
myelination
in
the
nur
7
mouse
model
of
Canavan
disease
.
Provision
of
the
odd
carbon
triglyceride
triheptanoin
to
neonatal
nur
7
mice
reduced
oxidative
stress
,
promoted
long
-term
oligodendrocyte
survival
,
and
increased
myelin
in
the
brain
.
Improvements
in
oligodendrocyte
survival
and
myelination
were
associated
with
a
highly
significant
reduction
in
spongiform
degeneration
and
improved
motor
function
in
triheptanoin
treated
mice
.
Initiation
of
triheptanoin
treatment
in
older
animals
resulted
in
markedly
more
modest
effects
on
these
same
pathological
indices
,
indicating
a
window
of
therapeutic
intervention
that
corresponds
with
developmental
myelination
.
These
results
support
the
targeting
of
oxidative
integrity
at
early
stages
of
Canavan
disease
,
and
provide
a
foundation
for
the
clinical
development
of
a
non-invasive
dietary
triheptanoin
treatment
regimen
.
Diseases
Validation
Diseases presenting
"indicating a window of therapeutic intervention that corresponds with developmental myelination"
symptom
canavan disease
You can validate or delete this automatically detected symptom
Validate the Symptom
Delete the Symptom