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Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.
[wiskott-aldrich syndrome]
Gene
transfer
into
the
hematopoietic
stem
cell
has
shown
curative
potential
for
a
variety
of
hematological
disorders
.
Primary
immunodeficiency
diseases
have
led
to
the
way
in
this
field
of
gene
therapy
as
an
example
and
a
model
.
Clinical
results
from
the
past
15
years
have
shown
that
significant
improvement
and
even
cure
can
be
achieved
for
diseases
such
as
X-
linked
severe
combined
immunodeficiency
,
adenosine
deaminase
deficiency
,
chronic
granulomatous
disease
and
Wiskott-
Aldrich
syndrome
.
Unfortunately
,
with
the
initial
clear
clinical
benefits
,
the
first
serious
complications
of
gene
therapy
have
also
occurred
.
In
a
significant
number
of
patients
treated
using
vectors
based
on
murine
gamma-retroviruses
and
carrying
powerful
viral
enhancer
elements
,
insertional
oncogenesis
events
have
resulted
in
acute
leukemias
that
,
in
some
cases
,
have
had
fatal
outcomes
.
These
serious
adverse
events
have
sparked
a
revision
of
the
assessment
of
risks
and
benefits
of
integrating
gene
transfer
for
hematological
diseases
and
prompted
the
development
and
application
of
new
generations
of
viral
vectors
with
recognized
superior
safety
characteristics
.
This
review
summarizes
the
clinical
experience
of
gene
therapy
for
primary
immunodeficiencies
and
discusses
the
likely
avenues
of
progress
in
the
future
development
of
this
expanding
field
of
clinical
investigations
.
Diseases
Validation
Diseases presenting
"immunodeficiency"
symptom
adrenal incidentaloma
allergic bronchopulmonary aspergillosis
cushing syndrome
dracunculiasis
hirschsprung disease
hodgkin lymphoma, classical
homocystinuria without methylmalonic aciduria
kabuki syndrome
legionellosis
malignant atrophic papulosis
oculocutaneous albinism
omenn syndrome
papillon-lefèvre syndrome
primary effusion lymphoma
primary hyperoxaluria type 1
pyomyositis
severe combined immunodeficiency
sneddon syndrome
werner syndrome
wiskott-aldrich syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
This symptom has already been validated
