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Physiological and tissue-specific vectors for treatment of inherited diseases.
[junctional epidermolysis bullosa]
After
more
than
1500
gene
therapy
clinical
trials
in
the
past
two
decades
,
the
overall
conclusion
is
that
for
gene
therapy
(
GT
)
to
be
successful
,
the
vector
systems
must
still
be
improved
in
terms
of
delivery
,
expression
and
safety
.
The
recent
development
of
more
efficient
and
stable
vector
systems
has
created
great
expectations
for
the
future
of
GT
.
Impressive
results
were
obtained
in
three
primary
immunodeficiencies
and
other
inherited
diseases
such
as
congenital
blindness
,
adrenoleukodystrophy
or
junctional
epidermolysis
bullosa
.
However
,
the
development
of
leukemia
in
five
children
included
in
the
GT
clinical
trials
for
X-
linked
severe
combined
immunodeficiency
and
the
silencing
of
the
therapeutic
gene
in
the
chronic
granulomatous
disease
clearly
showed
the
importance
of
improving
safety
and
efficiency
.
In
this
review
,
we
focus
on
the
main
strategies
available
to
achieve
physiological
or
tissue-
specific
expression
of
therapeutic
transgenes
and
discuss
the
importance
of
controlling
transgene
expression
to
improve
safety
.
We
propose
that
tissue-
specific
and
/
or
physiological
viral
vectors
offer
the
best
balance
between
efficiency
and
safety
and
will
be
the
tools
of
choice
for
future
clinical
trials
in
GT
of
inherited
diseases
.
Diseases
Validation
Diseases presenting
"future clinical trials"
symptom
alexander disease
epidermolysis bullosa simplex
erythropoietic protoporphyria
junctional epidermolysis bullosa
krabbe disease
proteus syndrome
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