-
1
-
-
0036446911
-
Widespread dispersal of cholera toxin subunit b to brain and spinal cord neurons following systemic delivery
-
Alisky JM, van de Wetering CI, and Davidson BL. Widespread dispersal of cholera toxin subunit b to brain and spinal cord neurons following systemic delivery. Exp Neurol 178: 139-146, 2002.
-
(2002)
Exp Neurol
, vol.178
, pp. 139-146
-
-
Alisky, J.M.1
van de Wetering, C.I.2
Davidson, B.L.3
-
2
-
-
33750716074
-
TDP-43 is a component of ubiquitin-positive tau-negative inclusions in frontotemporal lobar degeneration and amyotrophic lateral sclerosis
-
Arai T, Hasegawa M, Akiyama H, Ikeda K, Nonaka T, Mori H, Mann D, Tsuchiya K, Yoshida M, Hashizume Y, and Oda T. TDP-43 is a component of ubiquitin-positive tau-negative inclusions in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Biochem Biophys Res Commun 351: 602-611, 2006.
-
(2006)
Biochem Biophys Res Commun
, vol.351
, pp. 602-611
-
-
Arai, T.1
Hasegawa, M.2
Akiyama, H.3
Ikeda, K.4
Nonaka, T.5
Mori, H.6
Mann, D.7
Tsuchiya, K.8
Yoshida, M.9
Hashizume, Y.10
Oda, T.11
-
3
-
-
2642526164
-
VEGF delivery with retrogradely transported lenti-vector prolongs survival in a mouse ALS model
-
Azzouz M, Ralph GS, Storkebaum E, Walmsley LE, Mitrophanous KA, Kingsman SM, Carmeliet P, and Mazarakis ND. VEGF delivery with retrogradely transported lenti-vector prolongs survival in a mouse ALS model. Nature 429: 413-417, 2004.
-
(2004)
Nature
, vol.429
, pp. 413-417
-
-
Azzouz, M.1
Ralph, G.S.2
Storkebaum, E.3
Walmsley, L.E.4
Mitrophanous, K.A.5
Kingsman, S.M.6
Carmeliet, P.7
Mazarakis, N.D.8
-
4
-
-
36348929875
-
One-year expression from high-capacity adenoviral vectors in the brains of animals with pre-existing anti-adenoviral immunity: Clinical implications
-
Barcia C, Jimenez-Dalmaroni M, Kroeger KM, Puntel M, Rapaport AJ, Larocque D, King GD, Johnson SA, Liu C, Xiong W, Candolfi M, Mondkar S, Ng P, Palmer D, Castro MG, and Lowenstein PR. One-year expression from high-capacity adenoviral vectors in the brains of animals with pre-existing anti-adenoviral immunity: Clinical implications Mol Ther 15: 2154-2163, 2007.
-
(2007)
Mol Ther
, vol.15
, pp. 2154-2163
-
-
Barcia, C.1
Jimenez-Dalmaroni, M.2
Kroeger, K.M.3
Puntel, M.4
Rapaport, A.J.5
Larocque, D.6
King, G.D.7
Johnson, S.A.8
Liu, C.9
Xiong, W.10
Candolfi, M.11
Mondkar, S.12
Ng, P.13
Palmer, D.14
Castro, M.G.15
Lowenstein, P.R.16
-
5
-
-
33749056809
-
ALS: A disease of motor neurons and their nonneuronal neighbors
-
Boillee S, Van de Velde C, and Cleveland DW. ALS: A disease of motor neurons and their nonneuronal neighbors. Neuron 52: 39-59, 2006.
-
(2006)
Neuron
, vol.52
, pp. 39-59
-
-
Boillee, S.1
Van de Velde, C.2
Cleveland, D.W.3
-
6
-
-
33744798774
-
Onset and progression in inherited ALS determined by motor neurons and microglia
-
Boillee S, Yamanaka K, Lobsiger CS, Copeland NG, Jenkins NA, Kassiotis G, Kollias G, and Cleveland DW. Onset and progression in inherited ALS determined by motor neurons and microglia. Science 312: 1389-1392, 2006.
-
(2006)
Science
, vol.312
, pp. 1389-1392
-
-
Boillee, S.1
Yamanaka, K.2
Lobsiger, C.S.3
Copeland, N.G.4
Jenkins, N.A.5
Kassiotis, G.6
Kollias, G.7
Cleveland, D.W.8
-
7
-
-
0028813380
-
Superoxide dismutase 1 subunits with mutations linked to familial amyotrophic lateral sclerosis do not affect wild-type subunit function
-
Borchelt DR, Guarnieri M, Wong PC, Lee MK, Slunt HS, Xu Z-S, Sisodia SS, Price DL, and Cleveland DW. Superoxide dismutase 1 subunits with mutations linked to familial amyotrophic lateral sclerosis do not affect wild-type subunit function. J Biol Chem 270: 3234-3238, 1995.
-
(1995)
J Biol Chem
, vol.270
, pp. 3234-3238
-
-
Borchelt, D.R.1
Guarnieri, M.2
Wong, P.C.3
Lee, M.K.4
Slunt, H.S.5
Xu, Z.-S.6
Sisodia, S.S.7
Price, D.L.8
Cleveland, D.W.9
-
8
-
-
0027965073
-
Superoxide dismutase 1 with mutations linked to familial amyotrophic lateral sclerosis possesses significant activity
-
Borchelt DR, Lee MK, Slunt HS, Guarnieri M, Xu ZS, Wong PC, Brown RHJ, Price DL, Sisodia SS, and Cleveland DW. Superoxide dismutase 1 with mutations linked to familial amyotrophic lateral sclerosis possesses significant activity. Proc Natl Acad Sci USA 91: 8292-8296, 1994.
-
(1994)
Proc Natl Acad Sci USA
, vol.91
, pp. 8292-8296
-
-
Borchelt, D.R.1
Lee, M.K.2
Slunt, H.S.3
Guarnieri, M.4
Xu, Z.S.5
Wong, P.C.6
Brown, R.H.J.7
Price, D.L.8
Sisodia, S.S.9
Cleveland, D.W.10
-
10
-
-
0032544674
-
Aggregation and motor neuron toxicity of an ALS-linked SOD1 mutant independent from wild type SOD1
-
Bruijn LI, Houseweart MK, Kato S, Anderson KL, Anderson SD, Ohama E, Reaume AG, Scott RW, and Cleveland DW. Aggregation and motor neuron toxicity of an ALS-linked SOD1 mutant independent from wild type SOD1. Science 281: 1851-4, 1998.
-
(1998)
Science
, vol.281
, pp. 1851-1854
-
-
Bruijn, L.I.1
Houseweart, M.K.2
Kato, S.3
Anderson, K.L.4
Anderson, S.D.5
Ohama, E.6
Reaume, A.G.7
Scott, R.W.8
Cleveland, D.W.9
-
11
-
-
0035859929
-
Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems
-
Caplen NJ, Parrish S, Imani F, Fire A, and Morgan RA. Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems. Proc Natl Acad Sci USA 98: 9742-9847, 2001.
-
(2001)
Proc Natl Acad Sci USA
, vol.98
, pp. 9742-9847
-
-
Caplen, N.J.1
Parrish, S.2
Imani, F.3
Fire, A.4
Morgan, R.A.5
-
12
-
-
0028933344
-
Neuropathological changes in two lines of mice carrying a transgene for mutant human Cu, Zn SOD, and in mice overexpressing wild type human SOD: A model of familial amyotrophic lateral sclerosis (FALS)
-
Dal Canto MC and Gurney ME. Neuropathological changes in two lines of mice carrying a transgene for mutant human Cu, Zn SOD, and in mice overexpressing wild type human SOD: A model of familial amyotrophic lateral sclerosis (FALS). Brain Res 676: 25-40, 1995.
-
(1995)
Brain Res
, vol.676
, pp. 25-40
-
-
Dal Canto, M.C.1
Gurney, M.E.2
-
13
-
-
33847645080
-
RNA interference: A tool for querying nervous system function and an emerging therapy
-
Davidson BL and Boudreau RL. RNA interference: A tool for querying nervous system function and an emerging therapy. Neuron 53: 781-788, 2007.
-
(2007)
Neuron
, vol.53
, pp. 781-788
-
-
Davidson, B.L.1
Boudreau, R.L.2
-
14
-
-
33646466296
-
Conversion to the amyotrophic lateral sclerosis phenotype is associated with intermolecular linked insoluble aggregates of SOD1 in mitochondria
-
Deng H-X, Shi Y, Furukawa Y, Zhai H, Fu R, Liu E, Gorrie GH, Khan MS, Hung W-Y, Bigio EH, Lukas T, Dal Canto MC, O'Halloran TV, and Siddique T. Conversion to the amyotrophic lateral sclerosis phenotype is associated with intermolecular linked insoluble aggregates of SOD1 in mitochondria. Proc Natl Acad Sci USA 103: 7142-7147, 2006.
-
(2006)
Proc Natl Acad Sci USA
, vol.103
, pp. 7142-7147
-
-
Deng, H.-X.1
Shi, Y.2
Furukawa, Y.3
Zhai, H.4
Fu, R.5
Liu, E.6
Gorrie, G.H.7
Khan, M.S.8
Hung, W.-Y.9
Bigio, E.H.10
Lukas, T.11
Dal Canto, M.C.12
O'Halloran, T.V.13
Siddique, T.14
-
15
-
-
36749033738
-
Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits
-
DiFiglia M, Sena-Esteves M, Chase K, Sapp E, Pfister E, Sass M, Yoder J, Reeves P, Pandey RK, Rajeev KG, Manoharan M, Sah DWY, Zamore PD, and Aronin N. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits. Proc Natl Acad Sci USA 104: 17204-17209, 2007.
-
(2007)
Proc Natl Acad Sci USA
, vol.104
, pp. 17204-17209
-
-
DiFiglia, M.1
Sena-Esteves, M.2
Chase, K.3
Sapp, E.4
Pfister, E.5
Sass, M.6
Yoder, J.7
Reeves, P.8
Pandey, R.K.9
Rajeev, K.G.10
Manoharan, M.11
Sah, D.W.Y.12
Zamore, P.D.13
Aronin, N.14
-
16
-
-
0242379146
-
Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis
-
Ding H, Schwarz DS, Keene A, Affar el B, Fenton L, Xia X, Shi Y, Zamore PD, and Xu Z. Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis. Aging Cell 2: 209-217, 2003.
-
(2003)
Aging Cell
, vol.2
, pp. 209-217
-
-
Ding, H.1
Schwarz, D.S.2
Keene, A.3
Affar el, B.4
Fenton, L.5
Xia, X.6
Shi, Y.7
Zamore, P.D.8
Xu, Z.9
-
17
-
-
0035942736
-
Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
-
Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, and Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411: 494-498., 2001.
-
(2001)
Nature
, vol.411
, pp. 494-498
-
-
Elbashir, S.M.1
Harborth, J.2
Lendeckel, W.3
Yalcin, A.4
Weber, K.5
Tuschl, T.6
-
18
-
-
0034084338
-
Induction of apoptosis by the dsRNA-dependent protein kinase (PKR): Mechanism of action
-
Gil J and Esteban M. Induction of apoptosis by the dsRNA-dependent protein kinase (PKR): mechanism of action. Apoptosis 5: 107-114., 2000.
-
(2000)
Apoptosis
, vol.5
, pp. 107-114
-
-
Gil, J.1
Esteban, M.2
-
19
-
-
0028284779
-
Motor neuron degeneration in mice that express a human Cu, Zn superoxide dismutase
-
Gurney ME, Pu H, Chiu AY, Dal Canto MC, Polchow CY, Alexander DD, Caliendo J, Hentati A, Kwon YW, Deng H-X, Chen W, Zhai P, Sufit RL, and Siddique T. Motor neuron degeneration in mice that express a human Cu, Zn superoxide dismutase. Science 264: 1772-1775, 1994.
-
(1994)
Science
, vol.264
, pp. 1772-1775
-
-
Gurney, M.E.1
Pu, H.2
Chiu, A.Y.3
Dal Canto, M.C.4
Polchow, C.Y.5
Alexander, D.D.6
Caliendo, J.7
Hentati, A.8
Kwon, Y.W.9
Deng, H.-X.10
Chen, W.11
Zhai, P.12
Sufit, R.L.13
Siddique, T.14
-
20
-
-
20244378556
-
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
-
Harper SQ, Staber PD, He X, Eliason SL, Martins IH, Mao Q, Yang L, Kotin RM, Paulson HL, and Davidson BL. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci USA 102: 5820-5825, 2005.
-
(2005)
Proc Natl Acad Sci USA
, vol.102
, pp. 5820-5825
-
-
Harper, S.Q.1
Staber, P.D.2
He, X.3
Eliason, S.L.4
Martins, I.H.5
Mao, Q.6
Yang, L.7
Kotin, R.M.8
Paulson, H.L.9
Davidson, B.L.10
-
21
-
-
0344240348
-
ALS- associated mutant SOD1G93A causes mitochondrial vacuolation by expansion of the intermembrane space and by involvement of SOD1 aggregation and peroxisomes
-
Higgins CM, Jung C, and Xu Z. ALS- associated mutant SOD1G93A causes mitochondrial vacuolation by expansion of the intermembrane space and by involvement of SOD1 aggregation and peroxisomes. BMC Neurosci 4: 16, 2003.
-
(2003)
BMC Neurosci
, vol.4
, pp. 16
-
-
Higgins, C.M.1
Jung, C.2
Xu, Z.3
-
22
-
-
38649139370
-
Efficient retrograde neuronal transduction utilizing self-complementary AAV1
-
Hollis ER, 2nd, Kadoya K, Hirsch M, Samulski RJ, and Tuszynski MH. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol Ther 16: 296-301, 2008.
-
(2008)
Mol Ther
, vol.16
, pp. 296-301
-
-
Hollis 2nd, E.R.1
Kadoya, K.2
Hirsch, M.3
Samulski, R.J.4
Tuszynski, M.H.5
-
23
-
-
0034520591
-
Human Cu/Zn superoxide dismutase (SOD1) overexpression in mice causes mitochondrial vacuolization, axonal degeneration, and premature motoneuron death and accelerates motoneuron disease in mice expressing a familial amyotrophic lateral sclerosis mutant SOD1
-
Jaarsma D, Haasdijk ED, Grashorn JAC, Hawkins R, van Duijn W, Verspaget HW, London J, and Holstege JC. Human Cu/Zn superoxide dismutase (SOD1) overexpression in mice causes mitochondrial vacuolization, axonal degeneration, and premature motoneuron death and accelerates motoneuron disease in mice expressing a familial amyotrophic lateral sclerosis mutant SOD1. Neurobiol Dis 7: 623-643, 2000.
-
(2000)
Neurobiol Dis
, vol.7
, pp. 623-643
-
-
Jaarsma, D.1
Haasdijk, E.D.2
Grashorn, J.A.C.3
Hawkins, R.4
van Duijn, W.5
Verspaget, H.W.6
London, J.7
Holstege, J.C.8
-
24
-
-
31544467869
-
Disulphide-reduced superoxide dismutase-1 in CNS of transgenic amyotrophic lateral sclerosis models
-
Jonsson PA, Graffmo KS, Andersen PM, Brannstrom T, Lindberg M, Oliveberg M, and Marklund SL. Disulphide-reduced superoxide dismutase-1 in CNS of transgenic amyotrophic lateral sclerosis models. Brain 129: 451-464, 2006.
-
(2006)
Brain
, vol.129
, pp. 451-464
-
-
Jonsson, P.A.1
Graffmo, K.S.2
Andersen, P.M.3
Brannstrom, T.4
Lindberg, M.5
Oliveberg, M.6
Marklund, S.L.7
-
25
-
-
42649120983
-
TARDBP mutations in individuals with sporadic and familial amyotrophic lateral sclerosis
-
Kabashi E, Valdmanis PN, Dion P, Spiegelman D, McConkey BJ, Velde CV, Bouchard J-P, Lacomblez L, Pochigaeva K, Salachas F, Pradat P-F, Camu W, Meininger V, Dupre N, and Rouleau GA. TARDBP mutations in individuals with sporadic and familial amyotrophic lateral sclerosis. Nat Genet 40: 572-574, 2008.
-
(2008)
Nat Genet
, vol.40
, pp. 572-574
-
-
Kabashi, E.1
Valdmanis, P.N.2
Dion, P.3
Spiegelman, D.4
McConkey, B.J.5
Velde, C.V.6
Bouchard, J.-P.7
Lacomblez, L.8
Pochigaeva, K.9
Salachas, F.10
Pradat, P.-F.11
Camu, W.12
Meininger, V.13
Dupre, N.14
Rouleau, G.A.15
-
26
-
-
0042528664
-
Retrograde viral delivery of IGF-1 prolongs sSurvival in a mouse ALS model
-
Kaspar BK, Llado J, Sherkat N, Rothstein JD, and Gage FH. Retrograde viral delivery of IGF-1 prolongs sSurvival in a mouse ALS model. Science 301: 839-842, 2003.
-
(2003)
Science
, vol.301
, pp. 839-842
-
-
Kaspar, B.K.1
Llado, J.2
Sherkat, N.3
Rothstein, J.D.4
Gage, F.H.5
-
27
-
-
18344369543
-
MicroRNA biogenesis: Coordinated cropping and dicing
-
Kim VN. MicroRNA biogenesis: Coordinated cropping and dicing. Nat Rev Mol Cell Biol 6: 376-385, 2005.
-
(2005)
Nat Rev Mol Cell Biol
, vol.6
, pp. 376-385
-
-
Kim, V.N.1
-
28
-
-
11844303500
-
Recent developments in gene transfer: Risk and ethics
-
Kimmelman J. Recent developments in gene transfer: risk and ethics. BMJ 330: 79-82, 2005.
-
(2005)
BMJ
, vol.330
, pp. 79-82
-
-
Kimmelman, J.1
-
29
-
-
42449102142
-
High-capacity adenovirus vector-mediated anti-glioma gene therapy in the presence of systemic anti-adenovirus immunity
-
King GD, Muhammad AKMG, Xiong W, Kroeger KM, Puntel M, Larocque D, Palmer D, Ng P, Lowenstein PR, and Castro MG. High-capacity adenovirus vector-mediated anti-glioma gene therapy in the presence of systemic anti-adenovirus immunity. J Virol 82: 4680-4684, 2008.
-
(2008)
J Virol
, vol.82
, pp. 4680-4684
-
-
King, G.D.1
Muhammad, A.K.M.G.2
Xiong, W.3
Kroeger, K.M.4
Puntel, M.5
Larocque, D.6
Palmer, D.7
Ng, P.8
Lowenstein, P.R.9
Castro, M.G.10
-
30
-
-
0033588966
-
Peripheral axotomy slows motoneuron degeneration in a transgenic mouse line expressing mutant SOD1 G93A
-
Kong J and Xu Z. Peripheral axotomy slows motoneuron degeneration in a transgenic mouse line expressing mutant SOD1 G93A. J Comp Neurol 412: 373-380, 1999.
-
(1999)
J Comp Neurol
, vol.412
, pp. 373-380
-
-
Kong, J.1
Xu, Z.2
-
31
-
-
58149269261
-
In vivo silencing of alpha-synuclein using naked siRNA
-
Lewis J, Melrose H, Bumcrot D, Hope A, Zehr C, Lincoln S, Braithwaite A, He Z, Ogholikhan S, Hinkle K, Kent C, Toudjarska I, Charisse K, Braich R, Pandey RK, Heckman M, Maraganore DM, Crook J, and Farrer MJ. In vivo silencing of alpha-synuclein using naked siRNA. Mol Neurodegener 3: 19, 2008.
-
(2008)
Mol Neurodegener
, vol.3
, pp. 19
-
-
Lewis, J.1
Melrose, H.2
Bumcrot, D.3
Hope, A.4
Zehr, C.5
Lincoln, S.6
Braithwaite, A.7
He, Z.8
Ogholikhan, S.9
Hinkle, K.10
Kent, C.11
Toudjarska, I.12
Charisse, K.13
Braich, R.14
Pandey, R.K.15
Heckman, M.16
Maraganore, D.M.17
Crook, J.18
Farrer, M.J.19
-
32
-
-
0036152214
-
Immunology of viral-vector-mediated gene transfer into the brain: An evolutionary and developmental perspective
-
Lowenstein PR. Immunology of viral-vector-mediated gene transfer into the brain: An evolutionary and developmental perspective. Trends Immunol 23: 23-30, 2002.
-
(2002)
Trends Immunol
, vol.23
, pp. 23-30
-
-
Lowenstein, P.R.1
-
33
-
-
35348984094
-
Immune responses to adenovirus and adeno- associated vectors used for gene therapy of brain diseases: The role of immunological synapses in understanding the cell biology of neuroimmune interactions
-
Lowenstein PR, Mandel RJ, Xiong WD, Kroeger K, and Castro MG. Immune responses to adenovirus and adeno- associated vectors used for gene therapy of brain diseases: the role of immunological synapses in understanding the cell biology of neuroimmune interactions. Curr Gene Ther 7: 347-660, 2007.
-
(2007)
Curr Gene Ther
, vol.7
, pp. 347-660
-
-
Lowenstein, P.R.1
Mandel, R.J.2
Xiong, W.D.3
Kroeger, K.4
Castro, M.G.5
-
34
-
-
0036022401
-
Adenovirus-mediated gene transfer of glial cell line-derived neurotrophic factor prevents motor neuron loss of transgenic model mice for amyotrophic lateral sclerosis
-
Manabe Y, Nagano I, Gazi MS, Murakami T, Shiote M, Shoji M, Kitagawa H, Setoguchi Y, and Abe K. Adenovirus-mediated gene transfer of glial cell line-derived neurotrophic factor prevents motor neuron loss of transgenic model mice for amyotrophic lateral sclerosis. Apoptosis 7: 329-334., 2002.
-
(2002)
Apoptosis
, vol.7
, pp. 329-334
-
-
Manabe, Y.1
Nagano, I.2
Gazi, M.S.3
Murakami, T.4
Shiote, M.5
Shoji, M.6
Kitagawa, H.7
Setoguchi, Y.8
Abe, K.9
-
35
-
-
4644336023
-
Revealing the world of RNA interference
-
Mello CC and Conte D. Revealing the world of RNA interference. Nature 431: 338-342, 2004.
-
(2004)
Nature
, vol.431
, pp. 338-342
-
-
Mello, C.C.1
Conte, D.2
-
36
-
-
33749632259
-
Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis
-
Neumann M, Sampathu DM, Kwong LK, Truax AC, Micsenyi MC, Chou TT, Bruce J, Schuck T, Grossman M, Clark CM, McCluskey LF, Miller BL, Masliah E, Mackenzie IR, Feldman H, Feiden W, Kretzschmar HA, Trojanowski JQ, and Lee VM. Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Science 314: 130-133, 2006.
-
(2006)
Science
, vol.314
, pp. 130-133
-
-
Neumann, M.1
Sampathu, D.M.2
Kwong, L.K.3
Truax, A.C.4
Micsenyi, M.C.5
Chou, T.T.6
Bruce, J.7
Schuck, T.8
Grossman, M.9
Clark, C.M.10
McCluskey, L.F.11
Miller, B.L.12
Masliah, E.13
Mackenzie, I.R.14
Feldman, H.15
Feiden, W.16
Kretzschmar, H.A.17
Trojanowski, J.Q.18
Lee, V.M.19
-
37
-
-
33747605320
-
Molecular biology of amyotrophic lateral sclerosis: Insights from genetics
-
Pasinelli P and Brown RH. Molecular biology of amyotrophic lateral sclerosis: insights from genetics. Nat Rev Neurosci 7: 710-723, 2006.
-
(2006)
Nat Rev Neurosci
, vol.7
, pp. 710-723
-
-
Pasinelli, P.1
Brown, R.H.2
-
38
-
-
20244381261
-
Silencing mutant SOD1 using RNAi protects against neuro-degeneration and extends survival in an ALS model
-
Ralph GS, Radcliffe PA, Day DM, Carthy JM, Leroux MA, Lee DCP, Wong L-F, Bilsland LG, Greensmith L, Kingsman SM, Mitrophanous KA, Mazarakis ND, and Azzouz M. Silencing mutant SOD1 using RNAi protects against neuro-degeneration and extends survival in an ALS model. Nat Med 11: 429-433, 2005.
-
(2005)
Nat Med
, vol.11
, pp. 429-433
-
-
Ralph, G.S.1
Radcliffe, P.A.2
Day, D.M.3
Carthy, J.M.4
Leroux, M.A.5
Lee, D.C.P.6
Wong, L.-F.7
Bilsland, L.G.8
Greensmith, L.9
Kingsman, S.M.10
Mitrophanous, K.A.11
Mazarakis, N.D.12
Azzouz, M.13
-
39
-
-
33845755467
-
Illuminating the silence: Understanding the structure and function of small RNAs
-
Rana TM. Illuminating the silence: Understanding the structure and function of small RNAs. Nat Rev Mol Cell Biol 8: 23-36, 2007.
-
(2007)
Nat Rev Mol Cell Biol
, vol.8
, pp. 23-36
-
-
Rana, T.M.1
-
40
-
-
17644383664
-
Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS
-
Raoul C, Abbas-Terki T, Bensadoun J-C, Guillot S, Haase G, Szulc J, Henderson CE, and Aebischer P. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med 11: 423-428, 2005.
-
(2005)
Nat Med
, vol.11
, pp. 423-428
-
-
Raoul, C.1
Abbas-Terki, T.2
Bensadoun, J.-C.3
Guillot, S.4
Haase, G.5
Szulc, J.6
Henderson, C.E.7
Aebischer, P.8
-
41
-
-
15844393658
-
Motor neurons in Cu/Zn superoxide dismutase-deficient mice develop normally but exhibit enhanced cell death after axonal injury
-
Reaume AG, Elliott JL, Hoffman EK, Kowall NW, Ferrante RJ, Siwek DF, Wilcox HM, Flood DG, Beal MF, Brown RH, Jr., Scott RW, and Snider WD. Motor neurons in Cu/Zn superoxide dismutase-deficient mice develop normally but exhibit enhanced cell death after axonal injury. Nat Genet 13: 43-47, 1996.
-
(1996)
Nat Genet
, vol.13
, pp. 43-47
-
-
Reaume, A.G.1
Elliott, J.L.2
Hoffman, E.K.3
Kowall, N.W.4
Ferrante, R.J.5
Siwek, D.F.6
Wilcox, H.M.7
Flood, D.G.8
Beal, M.F.9
Brown Jr., R.H.10
Scott, R.W.11
Snider, W.D.12
-
42
-
-
54749133105
-
Advances in helper-dependent adenoviral vector research
-
Segura MM, Alba R, Bosch A, and Chillon M. Advances in helper-dependent adenoviral vector research. Curr Gene Ther 8: 222-235, 2008.
-
(2008)
Curr Gene Ther
, vol.8
, pp. 222-235
-
-
Segura, M.M.1
Alba, R.2
Bosch, A.3
Chillon, M.4
-
43
-
-
0038121109
-
Specificity of short interfering RNA determined through gene expression signatures
-
Semizarov D, Frost L, Sarthy A, Kroeger P, Halbert DN, and Fesik SW. Specificity of short interfering RNA determined through gene expression signatures. Proc Natl Acad Sci USA 100: 6347-6352, 2003.
-
(2003)
Proc Natl Acad Sci USA
, vol.100
, pp. 6347-6352
-
-
Semizarov, D.1
Frost, L.2
Sarthy, A.3
Kroeger, P.4
Halbert, D.N.5
Fesik, S.W.6
-
44
-
-
0037213559
-
Mammalian RNAi for the masses
-
Shi Y. Mammalian RNAi for the masses. Trends Genet 19: 9-12, 2003.
-
(2003)
Trends Genet
, vol.19
, pp. 9-12
-
-
Shi, Y.1
-
45
-
-
27744588007
-
Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model
-
Singer O, Marr RA, Rockenstein E, Crews L, Coufal NG, Gage FH, Verma IM, and Masliah E. Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model. Nat Neurosci 8: 1343-1349, 2005.
-
(2005)
Nat Neurosci
, vol.8
, pp. 1343-1349
-
-
Singer, O.1
Marr, R.A.2
Rockenstein, E.3
Crews, L.4
Coufal, N.G.5
Gage, F.H.6
Verma, I.M.7
Masliah, E.8
-
46
-
-
45549099627
-
Gene transfer into neural cells in vitro using adenoviral vectors
-
Southgate T, Kroeger KM, Liu C, Lowenstein PR, and Castro MG. Gene transfer into neural cells in vitro using adenoviral vectors. Curr Protoc Neurosci Chapter 4: Unit 4 23, 2008.
-
(2008)
Curr Protoc Neurosci Chapter 4: Unit
, vol.4
, pp. 23
-
-
Southgate, T.1
Kroeger, K.M.2
Liu, C.3
Lowenstein, P.R.4
Castro, M.G.5
-
47
-
-
41149180753
-
TDP-43 Mutations in familial and sporadic amyotrophic lateral sclerosis
-
Sreedharan J, Blair IP, Tripathi VB, Hu X, Vance C, Rogelj B, Ackerley S, Durnall JC, Williams KL, Buratti E, Baralle F, de Belleroche J, Mitchell JD, Leigh PN, Al-Chalabi A, Miller CC, Nicholson G, and Shaw CE. TDP-43 Mutations in familial and sporadic amyotrophic lateral sclerosis. Science 319: 1668-1672, 2008.
-
(2008)
Science
, vol.319
, pp. 1668-1672
-
-
Sreedharan, J.1
Blair, I.P.2
Tripathi, V.B.3
Hu, X.4
Vance, C.5
Rogelj, B.6
Ackerley, S.7
Durnall, J.C.8
Williams, K.L.9
Buratti, E.10
Baralle, F.11
de Belleroche, J.12
Mitchell, J.D.13
Leigh, P.N.14
Al-Chalabi, A.15
Miller, C.C.16
Nicholson, G.17
Shaw, C.E.18
-
48
-
-
33646403729
-
Intrathecal long-term gene expression by self-complementary adeno- associated virus type 1 suitable for chronic pain studies in rats
-
Storek B, Harder NM, Banck MS, Wang C, McCarty DM, Janssen WG, Morrison JH, Walsh CE, and Beutler AS. Intrathecal long-term gene expression by self-complementary adeno- associated virus type 1 suitable for chronic pain studies in rats. Mol Pain 2: 4, 2006.
-
(2006)
Mol Pain
, vol.2
, pp. 4
-
-
Storek, B.1
Harder, N.M.2
Banck, M.S.3
Wang, C.4
McCarty, D.M.5
Janssen, W.G.6
Morrison, J.H.7
Walsh, C.E.8
Beutler, A.S.9
-
49
-
-
0034926344
-
Preexisting antiadenoviral immunity is not a barrier to efficient and stable transduction of the brain, mediated by novel high-capacity adenovirus vectors
-
Thomas CE, Schiedner G, Kochanek S, Castro MG, and Lowenstein PR. Preexisting antiadenoviral immunity is not a barrier to efficient and stable transduction of the brain, mediated by novel high-capacity adenovirus vectors. Hum Gen Ther 12: 839-846, 2001.
-
(2001)
Hum Gen Ther
, vol.12
, pp. 839-846
-
-
Thomas, C.E.1
Schiedner, G.2
Kochanek, S.3
Castro, M.G.4
Lowenstein, P.R.5
-
50
-
-
14644393684
-
Perspective: Machines for RNAi
-
Tomari Y and Zamore PD. Perspective: Machines for RNAi. Genes Dev. 19: 517-529, 2005.
-
(2005)
Genes Dev
, vol.19
, pp. 517-529
-
-
Tomari, Y.1
Zamore, P.D.2
-
51
-
-
0036431701
-
Insect cells as a factory to produce adeno- associated virus type 2 vectors
-
Urabe M, Ding C, and Kotin RM. Insect cells as a factory to produce adeno- associated virus type 2 vectors. Hum Gen Ther 13: 1935-1943, 2002.
-
(2002)
Hum Gen Ther
, vol.13
, pp. 1935-1943
-
-
Urabe, M.1
Ding, C.2
Kotin, R.M.3
-
52
-
-
62549159967
-
Tailoring the AAV vector capsid for gene therapy
-
Vandenberghe LH, Wilson JM, and Gao G. Tailoring the AAV vector capsid for gene therapy. Gene Ther 16: 311-319, 2009.
-
(2009)
Gene Ther
, vol.16
, pp. 311-319
-
-
Vandenberghe, L.H.1
Wilson, J.M.2
Gao, G.3
-
53
-
-
0042525989
-
Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain
-
Wang C, Wang CM, Clark KR, and Sferra TJ. Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain. Gene Ther 10: 1528-1534, 2003.
-
(2003)
Gene Ther
, vol.10
, pp. 1528-1534
-
-
Wang, C.1
Wang, C.M.2
Clark, K.R.3
Sferra, T.J.4
-
54
-
-
47049099443
-
Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression
-
Wang H, Ghosh A, Baigude H, Yang C-S, Qiu L, Xia X, Zhou H, Rana TM, and Xu Z. Therapeutic gene silencing delivered by a chemically modified small interfering RNA against mutant SOD1 slows amyotrophic lateral sclerosis progression. J Biol Chem 283: 15845-15852, 2008.
-
(2008)
J Biol Chem
, vol.283
, pp. 15845-15852
-
-
Wang, H.1
Ghosh, A.2
Baigude, H.3
Yang, C.-S.4
Qiu, L.5
Xia, X.6
Zhou, H.7
Rana, T.M.8
Xu, Z.9
-
55
-
-
0036076642
-
Fibrillar inclusions and motor neuron degeneration in transgenic mice expressing superoxide dismutase 1 with a disrupted copper-binding site
-
Wang J, Xu G, Gonzales V, Coonfield M, Fromholt D, Copeland NG, Jenkins NA, and Borchelt DR. Fibrillar inclusions and motor neuron degeneration in transgenic mice expressing superoxide dismutase 1 with a disrupted copper-binding site. Neurobiol Dis 10: 128-138, 2002.
-
(2002)
Neurobiol Dis
, vol.10
, pp. 128-138
-
-
Wang, J.1
Xu, G.2
Gonzales, V.3
Coonfield, M.4
Fromholt, D.5
Copeland, N.G.6
Jenkins, N.A.7
Borchelt, D.R.8
-
57
-
-
0029053881
-
An adverse property of a familial ALS-linked SOD1 mutation causes motor neuron disease characterized by vacuolar degeneration of mitochondria
-
Wong PC, Pardo CA, Borchelt DR, Lee MK, Copeland NG, Jenkins NA, Sisodia SS, Cleveland DW, and Price DL. An adverse property of a familial ALS-linked SOD1 mutation causes motor neuron disease characterized by vacuolar degeneration of mitochondria. Neuron 14: 1105-1116, 1995.
-
(1995)
Neuron
, vol.14
, pp. 1105-1116
-
-
Wong, P.C.1
Pardo, C.A.2
Borchelt, D.R.3
Lee, M.K.4
Copeland, N.G.5
Jenkins, N.A.6
Sisodia, S.S.7
Cleveland, D.W.8
Price, D.L.9
-
58
-
-
4043057946
-
RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia
-
Xia H, Mao Q, Eliason SL, Harper SQ, Martins IH, Orr HT, Paulson HL, Yang L, Kotin RM, and Davidson BL. RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia. Nat Med 10: 816-820, 2004.
-
(2004)
Nat Med
, vol.10
, pp. 816-820
-
-
Xia, H.1
Mao, Q.2
Eliason, S.L.3
Harper, S.Q.4
Martins, I.H.5
Orr, H.T.6
Paulson, H.L.7
Yang, L.8
Kotin, R.M.9
Davidson, B.L.10
-
59
-
-
33747201641
-
Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo
-
Xia X, Zhou H, Huang Y, and Xu Z. Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo. Neurobiol Dis 23: 578-586, 2006.
-
(2006)
Neurobiol Dis
, vol.23
, pp. 578-586
-
-
Xia, X.1
Zhou, H.2
Huang, Y.3
Xu, Z.4
-
60
-
-
13244284793
-
An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu,Zn superoxide dismutase
-
Xia XG, Zhou H, Zhou S, Yu Y, Wu R, and Xu Z. An RNAi strategy for treatment of amyotrophic lateral sclerosis caused by mutant Cu,Zn superoxide dismutase. J Neurochem 92: 362-367, 2005.
-
(2005)
J Neurochem
, vol.92
, pp. 362-367
-
-
Xia, X.G.1
Zhou, H.2
Zhou, S.3
Yu, Y.4
Wu, R.5
Xu, Z.6
-
61
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno- associated virus vector
-
Xiao X, Li J, and Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno- associated virus vector. J Virol 70: 8098-8108, 1996.
-
(1996)
J Virol
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
62
-
-
33646006698
-
Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses
-
Xiong W, Goverdhana S, Sciascia SA, Candolfi M, Zirger JM, Barcia C, Curtin JF, King GD, Jaita G, Liu C, Kroeger K, Agadjanian H, Medina-Kauwe L, Palmer D, Ng P, Lowenstein PR, and Castro MG. Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses. J Virol 80: 27-37, 2006.
-
(2006)
J Virol
, vol.80
, pp. 27-37
-
-
Xiong, W.1
Goverdhana, S.2
Sciascia, S.A.3
Candolfi, M.4
Zirger, J.M.5
Barcia, C.6
Curtin, J.F.7
King, G.D.8
Jaita, G.9
Liu, C.10
Kroeger, K.11
Agadjanian, H.12
Medina-Kauwe, L.13
Palmer, D.14
Ng, P.15
Lowenstein, P.R.16
Castro, M.G.17
-
63
-
-
28644450043
-
An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi
-
Zhou H, Xia XG, and Xu Z. An RNA polymerase II construct synthesizes short-hairpin RNA with a quantitative indicator and mediates highly efficient RNAi. Nucl Acids Res 33: e62, 2005.
-
(2005)
Nucl Acids Res
, vol.33
-
-
Zhou, H.1
Xia, X.G.2
Xu, Z.3
|