Rare Diseases Symptoms Automatic Extraction
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A random Abstract
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Recent gene therapy advancements for neurological diseases.
[canavan disease]
The
past
few
years
have
seen
rapid
advancements
in
vector-mediated
gene
transfer
to
the
nervous
system
and
modest
successes
in
human
gene
therapy
trials
.
The
purpose
of
this
review
is
to
describe
commonly-used
viral
gene
transfer
vectors
and
recent
advancements
towards
producing
meaningful
gene
-based
treatments
for
central
nervous
system
(
CNS
)
disorders
.
Gene
therapy
trials
for
Canavan
disease
,
Batten
disease
,
adrenoleukodystrophy
,
and
Parkinson
's
disease
are
discussed
to
illustrate
the
current
state
of
clinical
gene
transfer
to
the
CNS
.
Preclinical
studies
are
under
way
for
a
number
of
diseases
,
primarily
lysosomal
storage
disorders
,
using
a
newer
generation
of
vectors
and
delivery
strategies
.
Relevant
studies
in
animal
models
are
highlighted
for
Mucopolysaccharidosis
IIIB
and
Krabbe
disease
to
provide
a
prelude
for
what
can
be
expected
in
the
coming
years
for
human
gene
transfer
trials
,
using
recent
advancements
in
gene
transfer
technology
.
In
conclusion
,
recent
improvements
in
CNS
gene
transfer
technology
are
expected
to
significantly
increase
the
degree
of
disease
rescue
in
future
CNS-directed
clinical
trials
,
exceeding
the
modest
clinical
successes
that
have
been
observed
so
far
.
Diseases
Validation
Diseases presenting
"primarily lysosomal storage disorders"
symptom
canavan disease
krabbe disease
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