Biomedical Engineering Reference
In-Depth Information
In dystroglycan KD zebrafish, we observed several defects that resemble human
congenital myopathies found in both DMD patients and
mdx
mice including (1) rapid,
progressive muscular degeneration, (2) immobility, (3) muscle defects, and (4) bent
spine (notochord). Although the zebrafish is phylogenically distant from humans, key
gene families have been shown to be highly conserved, and dystroglycan knockdown
performed by simple injection into wild-type animals is straightforward and highly
reproducible. An additional compelling advantage of this model organism is that a
battery of disease-specific zebrafish assays can be designed to assess drug effects.
ACKNOWLEDGMENT
This researchwas supported by a National Institutes of Health grant: 1R43AR055390.
REFERENCES
A
LLAMAND
V and C
AMPBELL
KP (2000). Animal models for muscular dystrophy: valuable tools for the
development of therapies. Hum Mol Genet 9(16): 2459-2467.
A
MALI
AA, L
IN
CJ, C
HEN
YH, W
ANG
WL, G
ONG
HY, R
EKHA
RD, L
U
JK, C
HEN
TT, and W
U
JL (2008).
Overexpression of Myostatin2 in zebrafish reduces the expression of dystrophin associated protein
complex (DAPC) which leads to muscle dystrophy. J Biomed Sci 15(5): 595-604.
A
RAISHI
K, S
ASAOKA
T, I
MAMURA
M, N
OGUCHI
S, H
AMA
H, W
AKABAYASHI
E, Y
OSHIDA
M, H
ORI
T, and O
ZAWA
E
(1999). Loss of the sarcoglycan complex and sarcospan leads tomuscular dystrophy in beta-sarcoglycan-
deficient mice. Hum Mol Genet 8(9): 1589-1598.
A
SSERETO
S, S
TRINGARA
S, S
OTGIA
F, B
ONUCCELLI
G, B
ROCCOLINI
A, P
EDEMONTE
M, T
RAVERSO
M, B
IANCHERI
R,
Z
ARA
F, B
RUNO
C, L
ISANTI
MP, and M
INETTI
C (2006). Pharmacological rescue of the dystrophin-gly-
coprotein complex in Duchenne and Becker skeletal muscle explants by proteasome inhibitor treatment.
Am J Physiol Cell Physiol 290(2): C577-C582.
B
ASSETT
DI, B
RYSON
-R
ICHARDSON
RJ, D
AGGETT
DF, G
AUTIER
P, K
EENAN
DG, and C
URRIE
PD (2003).
Dystrophin is required for the formation of stable muscle attachments in the zebrafish embryo.
Development 130(23): 5851-5860.
B
ASSETT
DI and C
URRIE
PD (2003). The zebrafish as a model for muscular dystrophy and congenital
myopathy. Hum Mol Genet 12 (Suppl 2): R265-R270.
B
ASSETT
DI and C
URRIE
PD (2004). Identification of a zebrafish model of muscular dystrophy. Clin Exp
Pharmacol Physiol 31(8): 537-540.
B
AUMEISTER
R and G
E
L (2002). The worm in us:
Caenorhabditis elegans
as a model of human disease.
Trends Biotechnol 20(4): 147-148.
B
EENAKKER
EA, F
OCK
JM, V
AN
T
OL
MJ, M
AURITS
NM, K
OOPMAN
HM, B
ROUWER
OF, and
VAN DER
H
OEVEN
JH
(2005). Intermittent prednisone therapy in Duchenne muscular dystrophy: a randomized controlled trial.
Arch Neurol 62(1): 128-132.
B
ESSOU
C, G
IUGIA
JB, F
RANKS
CJ, H
OLDEN
-D
YE
L, and S
EGALAT
L (1998). Mutations in the
Caenorhabditis
elegans
dystrophin-like gene dys-1 lead to hyperactivity and suggest a linkwith cholinergic transmission.
Neurogenetics 2(1): 61-72.
B
OGDANOVICH
S, K
RAG
TO, B
ARTON
ER, M
ORRIS
LD, W
HITTEMORE
LA, A
HIMA
RS, and K
HURANA
TS
(2002). Functional improvement of dystrophic muscle by myostatin blockade. Nature 420(6914):
418-421.
B
OGDANOVICH
S, P
ERKINS
KJ, K
RAG
TO, W
HITTEMORE
LA, and K
HURANA
TS (2005). Myostatin propeptide-
mediated amelioration of dystrophic pathophysiology. FASEB J 19(6): 543-549.
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