Rare Diseases Symptoms Automatic Extraction
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A random Abstract
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Clinical applications involving CNS gene transfer.
[canavan disease]
Diseases
of
the
central
nervous
system
(
CNS
)
have
traditionally
been
the
most
difficult
to
treat
by
traditional
pharmacological
methods
,
due
mostly
to
the
blood
-
brain
barrier
and
the
difficulties
associated
with
repeated
drug
administration
targeting
the
CNS
.
Viral
vector
gene
transfer
represents
a
way
to
permanently
provide
a
therapeutic
protein
within
the
nervous
system
after
a
single
administration
,
whether
this
be
a
gene
replacement
strategy
for
an
inherited
disorder
or
a
disease-modifying
protein
for
a
disease
such
as
Parkinson
's
.
Gene
therapy
approaches
for
CNS
disorders
has
evolved
considerably
over
the
last
two
decades
.
Although
a
breakthrough
treatment
has
remained
elusive
,
current
strategies
are
now
considerably
safer
and
potentially
much
more
effective
.
This
chapter
will
explore
the
past
,
current
,
and
future
status
of
CNS
gene
therapy
,
focusing
on
clinical
trials
utilizing
adeno-associated
virus
and
lentiviral
vectors
.
Diseases
Validation
Diseases presenting
"central nervous system"
symptom
22q11.2 deletion syndrome
adrenomyeloneuropathy
alexander disease
aniridia
aromatase deficiency
canavan disease
child syndrome
classical phenylketonuria
congenital toxoplasmosis
cowden syndrome
cushing syndrome
cystinuria
dracunculiasis
erdheim-chester disease
fabry disease
gm1 gangliosidosis
hereditary cerebral hemorrhage with amyloidosis
hirschsprung disease
hodgkin lymphoma, classical
kabuki syndrome
kallmann syndrome
kindler syndrome
krabbe disease
lamellar ichthyosis
legionellosis
liposarcoma
malignant atrophic papulosis
monosomy 21
neonatal adrenoleukodystrophy
phenylketonuria
proteus syndrome
scrub typhus
severe combined immunodeficiency
sneddon syndrome
triple a syndrome
von hippel-lindau disease
waldenström macroglobulinemia
well-differentiated liposarcoma
wiskott-aldrich syndrome
wolf-hirschhorn syndrome
x-linked adrenoleukodystrophy
zellweger syndrome
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