原创力文档Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy 英文参考文献.docVIP
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Allele-Specific Gene Silencing in Two Mouse Models of Autosomal Dominant Skeletal Myopathy 英文参考文献
Allele-SpecificGeneSilencinginTwoMouseModelsof
AutosomalDominantSkeletalMyopathy
RyanE.Loy,JohnD.Lueck,MohammedA.Mostajo-Radji,EllieM.Carrell,RobertT.Dirksen*
Department
of
Pharmacology
and
Physiology,
University
of
Rochester,
Rochester,
New
York,
United
States
of
America
Abstract
Weexplored
establishedmousemodelsoftheautosomaldominantly-inheritedmuscledisorders,MalignantHyperthermia(MH)and
CentralCoreDisease(CCD).CandidateASGSsweredesignedandvalidatedforefficacyandspecificityonryanodine
sexpressedinHEK293cellsusingRT-PCR-andconfocalmicroscopy-basedassays.In
t
h
e
potential
of
mutant
allele-specific
gene
silencing
(ASGS)
in
providing
therapeutic
benefit
in
two
s
i
R
N
A
receptor
(
R
y
R
1
)
c
D
N
A
m
i
ni
-
c
o
n
s
t
r
u
c
t
vivodeliveryof
noffootpads
establishedmousesof
sinemaximum
e(,
t
h
e
most
efficacious
identified
s
i
d
dCCD,respectively.TreatmentofIT/+miceresultedinamodestrescueof
R
N
h
A
e
s
t
i
e
n
r
t
o
o
zy
f
l
g
e
o
x
u
o
s
rdigitorum
r
b
r
e
v
i
(YS/
s
(
F
D
B
)
)and
m
R
u
y
s
r
c
1
l
e
s
w
a
s
a
(IT/)k
chieved
+
b
o
yinjection/
nmice,
Y524S/
+
+
I4895T/
+
e
l
e
c
t
r
o
p
o
r
a
t
i
o
o
f
4
–
6
m
on
t
h
o
l
R
y
1
n
c
k
-
i
m
od
e
l
r
M
H
with
c
o
r
e
%rescue)and
s
a
n
,
78%)
f
ligand-induced
Ca
2
+
d
in
the
d
e
f
i
c
i
t
t
h
a
t
3
8
m
a
g
n
i
t
ud
e
(
o
release
t
h
a
t
o
c
c
u
r
r
e
2+
absenceofachangeinthemagnitudeofelectrically-evokedCarelease.Comparedtothedifferencebetweenthecaffeine
2+
+
sensitivityofCareleaseinFDB
w
f
i
b
n
e
or
r
s
f
ma
r
o
m
Y
d
S
i
/
a
D
n
B
d
W
T
m
i
c
sfromefollowing2
etreatedwithSCRsiRNA
(
(
E
C50Mversus
:
1
.
1
m
4.4mM,
r
e
s
p
e
c
t
i
ve
l
y
)
,
c
af
f
ei
n
e
s
e
n
s
i
t
i
vi
t
y
a
s
l
i
z
e
n
F
f
i
b
e
r
Y
S
/+
m
i
c
E
C50:2.8mM)and4week(EC50:
2+
6.6mM)treatmentwithYSallele-specificsiRNA.Moreover,thetemperature-dependentincreasein
FDBfibersewasoWTlevelsafter2weeksoftreatmentwithYSallele-specificsiRNA.
/+
determinedbyltimePCR,edegreeoffunctionalrescue
/+/+micecorrelatedwellwiththe
r
e
s
t
i
n
g
C
a
o
b
s
e
r
v
e
d
i
n
f
r
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