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A modified γ-retrovirus vector for X-linked severe combined immunodeficiency.
[severe combined immunodeficiency]
In
previous
clinical
trials
involving
children
with
X-
linked
severe
combined
immunodeficiency
(
SCID
-X
1
)
,
a
Moloney
murine
leukemia
virus-based
γ-retrovirus
vector
expressing
interleukin-
2
receptor
γ-chain
(
γc
)
complementary
DNA
successfully
restored
immunity
in
most
patients
but
resulted
in
vector-induced
leukemia
through
enhancer-mediated
mutagenesis
in
25
%
of
patients
.
We
assessed
the
efficacy
and
safety
of
a
self-inactivating
retrovirus
for
the
treatment
of
SCID
-X
1
.
We
enrolled
nine
boys
with
SCID
-X
1
in
parallel
trials
in
Europe
and
the
United
States
to
evaluate
treatment
with
a
self-inactivating
(
SIN
)
γ-retrovirus
vector
containing
deletions
in
viral
enhancer
sequences
expressing
γc
(
SIN-γc
)
.
All
patients
received
bone
marrow-derived
CD
34
+
cells
transduced
with
the
SIN-γc
vector
,
without
preparative
conditioning
.
After
12
.
1
to
38
.
7
months
of
follow-up
,
eight
of
the
nine
children
were
still
alive
.
One
patient
died
from
an
overwhelming
adenoviral
infection
before
reconstitution
with
genetically
modified
T
cells
.
Of
the
remaining
eight
patients
,
seven
had
recovery
of
peripheral
-
blood
T
cells
that
were
functional
and
led
to
resolution
of
infections
.
The
patients
remained
healthy
thereafter
.
The
kinetics
of
CD
3
+
T
-
cell
recovery
was
not
significantly
different
from
that
observed
in
previous
trials
.
Assessment
of
insertion
sites
in
peripheral
blood
from
patients
in
the
current
trial
as
compared
with
those
in
previous
trials
revealed
significantly
less
clustering
of
insertion
sites
within
LMO
2
,
MECOM
,
and
other
lymphoid
proto-oncogenes
in
our
patients
.
This
modified
γ-retrovirus
vector
was
found
to
retain
efficacy
in
the
treatment
of
SCID
-X
1
.
The
long
-term
effect
of
this
therapy
on
leukemogenesis
remains
unknown
.
(
Funded
by
the
National
Institutes
of
Health
and
others
;
ClinicalTrials
.
gov
numbers
,
NCT
01410019
,
NCT
01175239
,
and
NCT
01129544
.
)
.
Diseases
Validation
Diseases presenting
"peripheral blood"
symptom
adrenomyeloneuropathy
allergic bronchopulmonary aspergillosis
aniridia
cohen syndrome
congenital toxoplasmosis
cutaneous mastocytosis
erdheim-chester disease
esophageal adenocarcinoma
esophageal squamous cell carcinoma
familial mediterranean fever
gm1 gangliosidosis
junctional epidermolysis bullosa
lamellar ichthyosis
monosomy 21
oligodontia
omenn syndrome
scrub typhus
severe combined immunodeficiency
typhoid
von hippel-lindau disease
waldenström macroglobulinemia
wiskott-aldrich syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
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