Rare Diseases Symptoms Automatic Extraction
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Long-term stability and safety of transgenic cultured epidermal stem cells in gene therapy of junctional epidermolysis bullosa.
[junctional epidermolysis bullosa]
We
report
a
long
-term
follow-up
(
6
.
5
years
)
of
a
phase
I
/
II
clinical
trial
envisaging
the
use
of
autologous
genetically
modified
cultured
epidermal
stem
cells
for
gene
therapy
of
junctional
epidermolysis
bullosa
,
a
devastating
genetic
skin
disease
.
The
critical
goals
of
the
trial
were
to
evaluate
the
safety
and
long
-term
persistence
of
genetically
modified
epidermis
.
A
normal
epidermal-dermal
junction
was
restored
and
the
regenerated
transgenic
epidermis
was
found
to
be
fully
functional
and
virtually
indistinguishable
from
a
normal
control
.
The
epidermis
was
sustained
by
a
discrete
number
of
long
-lasting
,
self-renewing
transgenic
epidermal
stem
cells
that
maintained
the
Â
memory
of
the
donor
site
,
whereas
the
vast
majority
of
transduced
transit-amplifying
progenitors
were
lost
within
the
first
few
months
after
grafting
.
These
data
pave
the
way
for
the
safe
use
of
epidermal
stem
cells
in
combined
cell
and
gene
therapy
for
genetic
skin
diseases
.
