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Epithelial stem cells in corneal regeneration and epidermal gene therapy.
[junctional epidermolysis bullosa]
Regenerative
medicine
refers
to
innovative
therapies
aimed
at
the
permanent
restoration
of
diseased
tissues
and
organs
.
Regeneration
of
self-renewing
tissues
requires
specific
adult
stem
cells
,
which
need
to
be
genetically
modified
to
correct
inherited
genetic
diseases
.
Cultures
of
epithelial
stem
cells
permanently
restore
severe
skin
and
mucosal
defects
,
and
genetically
corrected
epidermal
stem
cells
regenerate
a
normal
epidermis
in
patients
carrying
junctional
epidermolysis
bullosa
.
The
keratinocyte
stem
cell
is
therefore
the
only
cultured
stem
cell
used
both
in
cell
therapy
and
gene
therapy
clinical
protocols
.
Epithelial
stem
cell
identification
,
fate
and
molecular
phenotype
have
been
extensively
reviewed
,
but
not
in
relation
to
tissue
regeneration
.
In
this
paper
we
focus
on
the
localization
and
molecular
characterization
of
human
limbal
stem
cells
in
relation
to
corneal
regeneration
,
and
the
gene
therapy
of
genetic
skin
diseases
by
means
of
genetically
modified
epidermal
stem
cells
.
Diseases
Validation
Diseases presenting
"which need to be genetically modified to correct inherited genetic diseases"
symptom
junctional epidermolysis bullosa
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