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Acknowledgments
Elisabeth Dirren is supported by a fellowship from the Swiss
Foundation for Research on Muscle Diseases (FSRMM). This
work was also supported by the SMA Foundation and by the
SystemsX.ch project “Neurochoice.” The authors thank Viviane
Padrun and Fabienne Pidoux for their expert help in establishing
protocols for AAV production.
References
1. Ferraiuolo L, Kirby J, Grierson AJ, Sendtner
M, Shaw PJ (2011) Molecular pathways of
motor neuron injury in amyotrophic lateral
sclerosis. Nat Rev Neurol 7:616-630
2. Wee CD, Kong L, Sumner CJ (2010) The
genetics of spinal muscular atrophies. Curr
Opin Neurol 23:450-458
3. Van Den Bosch L (2011) Genetic rodent
models of amyotrophic lateral sclerosis. J
Biomed Biotechnol 2011:348765
4. Pun S, Santos AF, Saxena S, Xu L, Caroni P
(2006) Selective vulnerability and pruning of
phasic motoneuron axons in motoneuron dis-
ease alleviated by CNTF. Nat Neurosci
9:408-419
5. Boillee S, Vande Velde C, Cleveland DW
(2006) ALS: a disease of motor neurons and
their
11. Boillee S, Cleveland DW (2004) Gene therapy
for ALS delivers. Trends Neurosci 27:235-238
12. Hester ME, Foust KD, Kaspar RW, Kaspar BK
(2009) AAV as a gene transfer vector for the
treatment of neurological disorders: novel
treatment thoughts for ALS. Curr Gene Ther
9:428-433
13. Henriques A, Pitzer C, Dittgen T, Klugmann
M, Dupuis L, Schneider A (2011) CNS-
targeted viral delivery of G-CSF in an animal
model for ALS: improved effi cacy and preser-
vation of the neuromuscular unit. Mol Ther
19:284-292
14. Franz CK, Federici T, Yang J, Backus C, Oh
SS, Teng Q, Carlton E, Bishop KM, Gasmi M,
Bartus RT, Feldman EL, Boulis NM (2009)
Intraspinal cord delivery of IGF-I mediated by
adeno-associated virus 2 is neuroprotective in
a rat model of familial ALS. Neurobiol Dis
33:473-481
15. Kaspar BK, Llado J, Sherkat N, Rothstein JD,
Gage FH (2003) Retrograde viral delivery of
IGF-1 prolongs survival in a mouse ALS
model. Science 301:839-842
16. Lepore AC, Haenggeli C, Gasmi M, Bishop
KM, Bartus RT, Maragakis NJ, Rothstein JD
(2007) Intraparenchymal spinal cord delivery
of adeno-associated virus IGF-1 is protective
in the SOD1G93A model of ALS. Brain Res
1185:256-265
17. Azzouz M, Hottinger A, Paterna JC, Zurn
AD, Aebischer P, Bueler H (2000) Increased
motoneuron survival and improved neuro-
muscular function in transgenic ALS mice after
intraspinal injection of an adeno-associated
virus encoding Bcl-2. Hum Mol Genet 9:
803-811
18. Ralph GS, Radcliffe PA, Day DM, Carthy JM,
Leroux MA, Lee DC, Wong LF, Bilsland LG,
Greensmith L, Kingsman SM, Mitrophanous
KA, Mazarakis ND, Azzouz M (2005)
Silencing mutant SOD1 using RNAi protects
against neurodegeneration and extends
survival in an ALS model. Nat Med 11:
429-433
nonneuronal
neighbors.
Neuron
52:39-59
6. Ilieva H, Polymenidou M, Cleveland DW
(2009) Non-cell autonomous toxicity in neu-
rodegenerative disorders: ALS and beyond. J
Cell Biol 187:761-772
7. Boillee S, Yamanaka K, Lobsiger CS, Copeland
NG, Jenkins NA, Kassiotis G, Kollias G,
Cleveland DW (2006) Onset and progression
in inherited ALS determined by motor neu-
rons and microglia. Science 312:1389-1392
8. Clement AM, Nguyen MD, Roberts EA,
Garcia ML, Boillee S, Rule M, McMahon AP,
Doucette W, Siwek D, Ferrante RJ, Brown RH
Jr, Julien JP, Goldstein LS, Cleveland DW
(2003) Wild-type nonneuronal cells extend
survival of SOD1 mutant motor neurons in
ALS mice. Science 302:113-117
9. Lobsiger CS, Cleveland DW (2007) Glial cells
as intrinsic components of non-cell-
autonomous neurodegenerative disease. Nat
Neurosci 10:1355-1360
10. Yamanaka K, Chun SJ, Boillee S, Fujimori-
Tonou N, Yamashita H, Gutmann DH,
Takahashi R, Misawa H, Cleveland DW (2008)
Astrocytes as determinants of disease progres-
sion in inherited amyotrophic lateral sclerosis.
Nat Neurosci 11:251-253
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