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Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix.
[dystrophic epidermolysis bullosa]
Contrary
to
the
prevailing
professional
opinion
of
the
past
few
decades
,
recent
experimental
and
clinical
data
support
the
fact
that
protein
replacement
therapy
by
allogeneic
blood
and
marrow
transplantation
is
not
limited
to
freely
diffusible
molecules
such
as
enzymes
,
but
also
large
structural
proteins
such
as
collagens
.
A
prime
example
is
the
cross-correction
of
type
VII
collagen
deficiency
in
generalised
severe
recessive
dystrophic
epidermolysis
bullosa
,
in
which
blood
and
marrow
transplantation
can
attenuate
the
mucocutaneous
manifestations
of
the
disease
and
improve
patients
'
quality
of
life
.
Although
allogeneic
blood
and
marrow
transplantation
can
improve
the
integrity
of
the
skin
and
mucous
membranes
,
today
's
accomplishments
are
only
the
first
steps
on
the
long
pathway
to
cure
.
Future
strategies
will
be
built
on
the
lessons
learned
from
these
first
transplant
studies
.
Diseases
Validation
Diseases presenting
"past few decades"
symptom
achondroplasia
allergic bronchopulmonary aspergillosis
congenital adrenal hyperplasia
dystrophic epidermolysis bullosa
esophageal adenocarcinoma
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