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Positioning ganglioside D3 as an immunotherapeutic target in lymphangioleiomyomatosis.
[lymphangioleiomyomatosis]
Tumors
that
develop
in
lymphangioleiomyomatosis
(
LAM
)
as
a
consequence
of
biallelic
loss
of
TSC
1
or
TSC
2
gene
function
express
melanoma
differentiation
antigens
.
However
,
the
percentage
of
LAM
cells
expressing
these
melanosomal
antigens
is
limited
.
Here
,
we
report
the
overexpression
of
ganglioside
D
3
(
GD
3
)
in
LAM
.
GD
3
is
a
tumor
-associated
antigen
otherwise
found
in
melanoma
and
neuroendocrine
tumors
;
normal
expression
is
largely
restricted
to
neuronal
cells
in
the
brain
.
We
also
observed
markedly
reduced
serum
antibody
titers
to
GD
3
,
which
may
allow
for
a
population
of
GD
3
-
expressing
LAM
cells
to
expand
within
patients
.
This
is
supported
by
the
demonstrated
sensitivity
of
cultured
LAM
cells
to
complement
mediated
cytotoxicity
via
GD
3
antibodies
.
GD
3
can
serve
as
a
natural
killer
T
(
NKT
)
cell
antigen
when
presented
on
CD
1
d
molecules
expressed
on
professional
antigen-presenting
cells
.
Although
CD
1
d-expressing
monocyte
derivatives
were
present
in
situ
,
enhanced
NKT-cell
recruitment
to
LAM
lung
was
not
observed
.
Cultured
LAM
cells
retained
surface
expression
of
GD
3
over
several
passages
and
also
expressed
CD
1
d
,
implying
that
infiltrating
NKT
cells
can
be
directly
cytotoxic
toward
LAM
lung
lesions
.
Immunization
with
antibodies
to
GD
3
may
thus
be
therapeutic
in
LAM
,
and
enhancement
of
existing
NKT-cell
infiltration
may
be
effective
to
further
improve
antitumor
responses
.
Overall
,
we
hereby
establish
GD
3
as
a
suitable
target
for
immunotherapy
of
LAM
.
Diseases
Validation
Diseases presenting
"lymphangioleiomyomatosis"
symptom
lymphangioleiomyomatosis
proteus syndrome
This symptom has already been validated