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Gene therapy for wiskott-Aldrich syndrome.
[wiskott-aldrich syndrome]
The
Wiskott-
Aldrich
Syndrome
(
WAS
)
is
a
monogenic
X-
linked
primary
immunodeficiency
characterised
also
by
thrombocytopenia
,
eczema
,
and
a
high
susceptibility
to
develop
tumours
and
autoimmunity
.
WAS
patients
have
a
severely
reduced
life
expectancy
,
unless
they
undergo
a
successful
HLA-matched
haematopoietic
stem
cell
(
HSC
)
transplantation
.
However
,
several
WAS
patients
lack
a
compatible
donor
and
complications
,
such
as
autoimmunity
,
can
arise
in
a
significant
fraction
of
HSC
transplanted
patients
.
Administration
of
WAS
gene
-corrected
autologous
HSC
represents
an
alternative
therapeutic
approach
,
potentially
applicable
to
all
WAS
patients
.
To
this
aim
,
several
gene
therapy
approaches
for
WAS
using
initially
γ-retroviral
vectors
(
RVs
)
and
subsequently
HIV-based
lentiviral
vectors
(
LVs
)
have
been
developed
.
In
the
present
review
,
we
will
first
describe
the
results
of
the
preclinical
studies
conducted
in
the
murine
model
of
WAS
and
then
discuss
the
outcome
of
different
phase
I
/
II
clinical
trials
using
RV
or
LV-
transduced
HSC
.
Both
gene
therapy
approaches
led
to
restored
WASP
expression
,
correction
of
functional
defects
and
clinical
improvement
.
While
RV-mediated
gene
therapy
was
associated
with
a
high
occurrence
of
leukaemia
,
results
obtained
in
the
first
patients
treated
with
LV-based
HSC
gene
therapy
indicate
a
safer
risk-benefit
profile
.
Diseases
Validation
Diseases presenting
"a high occurrence of leukaemia"
symptom
wiskott-aldrich syndrome
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