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19. Raoul C, Abbas-Terki T, Bensadoun JC,
Guillot S, Haase G, Szulc J, Henderson CE,
Aebischer P (2005) Lentiviral-mediated silenc-
ing of SOD1 through RNA interference
retards disease onset and progression in a
mouse model of ALS. Nat Med 11:423-428
20. Burghes AH, Beattie CE (2009) Spinal mus-
cular atrophy: why do low levels of survival
motor neuron protein make motor neurons
sick? Nat Rev Neurosci 10:597-609
21. Passini MA, Cheng SH (2011) Prospects for
the gene therapy of spinal muscular atrophy.
Trends Mol Med 17:259-265
22. Le TT, Pham LT, Butchbach ME, Zhang HL,
Monani UR, Coovert DD, Gavrilina TO, Xing
L, Bassell GJ, Burghes AH (2005) SMNDelta7,
the major product of the centromeric survival
motor neuron (SMN2) gene, extends survival
in mice with spinal muscular atrophy and asso-
ciates with full-length SMN. Hum Mol Genet
14:845-857
23. Foust KD, Wang X, McGovern VL, Braun L,
Bevan AK, Haidet AM, Le TT, Morales PR,
Rich MM, Burghes AH, Kaspar BK (2010)
Rescue of the spinal muscular atrophy pheno-
type in a mouse model by early postnatal deliv-
ery of SMN. Nat Biotechnol 28:271-274
24. Passini MA, Bu J, Roskelley EM, Richards AM,
Sardi SP, O'Riordan CR, Klinger KW,
Shihabuddin LS, Cheng SH (2010) CNS-
targeted gene therapy improves survival and
motor function in a mouse model of spinal mus-
cular atrophy. J Clin Invest 120:1253-1264
25. Dominguez E, Marais T, Chatauret N,
Benkhelifa-Ziyyat S, Duque S, Ravassard P,
Carcenac R, Astord S, Pereira de Moura A, Voit
T, Barkats M (2011) Intravenous scAAV9 deliv-
ery of a codon-optimized SMN1 sequence res-
cues SMA mice. Hum Mol Genet 20:681-693
26. Valori CF, Ning K, Wyles M, Mead RJ,
Grierson AJ, Shaw PJ, Azzouz M (2010)
Systemic delivery of scAAV9 expressing SMN
prolongs survival in a model of spinal muscular
atrophy. Sci Transl Med 2:35ra42
27. Azzouz M, Ralph GS, Storkebaum E, Walmsley
LE, Mitrophanous KA, Kingsman SM,
Carmeliet P, Mazarakis ND (2004) VEGF
delivery with retrogradely transported lenti-
vector prolongs survival in a mouse ALS
model. Nature 429:413-417
28. Mentis GZ, Gravell M, Hamilton R, Shneider
NA, O'Donovan MJ, Schubert M (2006)
Transduction of motor neurons and muscle
fi bers by intramuscular injection of HIV-1-
based vectors pseudotyped with select rabies
virus glycoproteins. J Neurosci Methods
157:208-217
29. Foust KD, Nurre E, Montgomery CL,
Hernandez A, Chan CM, Kaspar BK (2009)
Intravascular AAV9 preferentially targets
neonatal neurons and adult astrocytes. Nat
Biotechnol 27:59-65
30. Duque S, Joussemet B, Riviere C, Marais T,
Dubreil L, Douar AM, Fyfe J, Moullier P,
Colle MA, Barkats M (2009) Intravenous
administration of self-complementary AAV9
enables transgene delivery to adult motor neu-
rons. Mol Ther 17:1187-1196
31. Miyake N, Miyake K, Yamamoto M, Hirai Y,
Shimada T (2011) Global gene transfer into
the CNS across the BBB after neonatal sys-
temic delivery of single-stranded AAV vectors.
Brain Res 1389:19-26
32.
Towne C, Schneider BL, Kieran D, Redmond
DE Jr, Aebischer P (2010) Effi cient transduc-
tion of non-human primate motor neurons
after intramuscular delivery of recombinant
AAV serotype 6. Gene Ther 17:141-146
33. Towne C, Setola V, Schneider BL, Aebischer P
(2011) Neuroprotection by gene therapy tar-
geting mutant SOD1 in individual pools of
motor neurons does not translate into therapeu-
tic benefi t in fALS mice. Mol Ther 19:274-283
34. Bevan AK, Duque S, Foust KD, Morales PR,
Braun L, Schmelzer L, Chan CM, McCrate M,
Chicoine LG, Coley BD, Porensky PN, Kolb
SJ, Mendell JR, Burghes AH, Kaspar BK
(2011) Systemic gene delivery in large species
for targeting spinal cord, brain, and peripheral
tissues for pediatric disorders. Mol Ther
19:1971-1980
35. Wu R, Wang H, Xia X, Zhou H, Liu C, Castro
M, Xu Z (2009) Nerve injection of viral vec-
tors effi ciently transfers transgenes into motor
neurons and delivers RNAi therapy against
ALS. Antioxid Redox Signal 11:1523-1534
36. Bru T, Salinas S, Kremer EJ (2010) An update
on canine adenovirus type 2 and its vectors.
Viruses 2:2134-2153
37. Salinas S, Bilsland LG, Henaff D, Weston AE,
Keriel A, Schiavo G, Kremer EJ (2009) CAR-
associated vesicular transport of an adenovirus in
motor neuron axons. PLoS Pathog 5:e1000442
38. Hollis ER 2nd, Kadoya K, Hirsch M, Samulski
RJ, Tuszynski MH (2008) Effi cient retrograde
neuronal transduction utilizing self-
complementary AAV1. Mol Ther 16:296-301
39. Hollis ER 2nd, Jamshidi P, Lorenzana AO,
Lee JK, Gray SJ, Samulski RJ, Zheng B,
Tuszynski MH (2010) Transient demyelin-
ation increases the effi ciency of retrograde
AAV transduction. Mol Ther 18:1496-1500
40. Miller TM, Kaspar BK, Kops GJ, Yamanaka K,
Christian LJ, Gage FH, Cleveland DW (2005)
Virus-delivered small RNA silencing sustains
strength in amyotrophic lateral sclerosis. Ann
Neurol 57:773-776
41. Towne C, Raoul C, Schneider BL, Aebischer P
(2008) Systemic AAV6 delivery mediating RNA
interference against SOD1: neuromuscular
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