-
1
-
-
2642512201
-
Clades of adeno-associated viruses are widely disseminated in human tissues
-
1 Gao, G, Vandenberghe, LH, Alvira, MR, Lu, Y, Calcedo, R, Zhou, X, et al. Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol 78 (2004), 6381–6388.
-
(2004)
J Virol
, vol.78
, pp. 6381-6388
-
-
Gao, G.1
Vandenberghe, L.H.2
Alvira, M.R.3
Lu, Y.4
Calcedo, R.5
Zhou, X.6
-
2
-
-
32944459983
-
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
-
2 Cearley, CN, Wolfe, JH, Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther 13 (2006), 528–537.
-
(2006)
Mol Ther
, vol.13
, pp. 528-537
-
-
Cearley, C.N.1
Wolfe, J.H.2
-
3
-
-
67649861393
-
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
-
3 Duque, S, Joussemet, B, Riviere, C, Marais, T, Dubreil, L, Douar, AM, et al. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther 17 (2009), 1187–1196.
-
(2009)
Mol Ther
, vol.17
, pp. 1187-1196
-
-
Duque, S.1
Joussemet, B.2
Riviere, C.3
Marais, T.4
Dubreil, L.5
Douar, A.M.6
-
4
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
4 Foust, KD, Nurre, E, Montgomery, CL, Hernandez, A, Chan, CM, Kaspar, BK, Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 27 (2009), 59–65.
-
(2009)
Nat Biotechnol
, vol.27
, pp. 59-65
-
-
Foust, K.D.1
Nurre, E.2
Montgomery, C.L.3
Hernandez, A.4
Chan, C.M.5
Kaspar, B.K.6
-
5
-
-
79955467779
-
Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors
-
5 Miyake, N, Miyake, K, Yamamoto, M, Hirai, Y, Shimada, T, Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors. Brain Res 1389 (2011), 19–26.
-
(2011)
Brain Res
, vol.1389
, pp. 19-26
-
-
Miyake, N.1
Miyake, K.2
Yamamoto, M.3
Hirai, Y.4
Shimada, T.5
-
6
-
-
77749249680
-
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
-
6 Foust, KD, Wang, X, McGovern, VL, Braun, L, Bevan, AK, Haidet, AM, et al. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 28 (2010), 271–274.
-
(2010)
Nat Biotechnol
, vol.28
, pp. 271-274
-
-
Foust, K.D.1
Wang, X.2
McGovern, V.L.3
Braun, L.4
Bevan, A.K.5
Haidet, A.M.6
-
7
-
-
77955602597
-
Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy
-
7 Valori, CF, Ning, K, Wyles, M, Mead, RJ, Grierson, AJ, Shaw, PJ, et al. Systemic delivery of scAAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy. Sci Transl Med, 2, 2010, 35ra42.
-
(2010)
Sci Transl Med
, vol.2
, pp. 35ra42
-
-
Valori, C.F.1
Ning, K.2
Wyles, M.3
Mead, R.J.4
Grierson, A.J.5
Shaw, P.J.6
-
8
-
-
78751700314
-
Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
-
8 Dominguez, E, Marais, T, Chatauret, N, Benkhelifa-Ziyyat, S, Duque, S, Ravassard, P, et al. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum Mol Genet 20 (2011), 681–693.
-
(2011)
Hum Mol Genet
, vol.20
, pp. 681-693
-
-
Dominguez, E.1
Marais, T.2
Chatauret, N.3
Benkhelifa-Ziyyat, S.4
Duque, S.5
Ravassard, P.6
-
9
-
-
84855760635
-
Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy
-
9 Glascock, JJ, Shababi, M, Wetz, MJ, Krogman, MM, Lorson, CL, Direct central nervous system delivery provides enhanced protection following vector mediated gene replacement in a severe model of spinal muscular atrophy. Biochem Biophys Res Commun 417 (2012), 376–381.
-
(2012)
Biochem Biophys Res Commun
, vol.417
, pp. 376-381
-
-
Glascock, J.J.1
Shababi, M.2
Wetz, M.J.3
Krogman, M.M.4
Lorson, C.L.5
-
10
-
-
84905456909
-
Systemic administration of a recombinant AAV1 vector encoding IGF-1 improves disease manifestations in SMA mice
-
10 Tsai, LK, Chen, CL, Ting, CH, Lin-Chao, S, Hwu, WL, Dodge, JC, et al. Systemic administration of a recombinant AAV1 vector encoding IGF-1 improves disease manifestations in SMA mice. Mol Ther 22 (2014), 1450–1459.
-
(2014)
Mol Ther
, vol.22
, pp. 1450-1459
-
-
Tsai, L.K.1
Chen, C.L.2
Ting, C.H.3
Lin-Chao, S.4
Hwu, W.L.5
Dodge, J.C.6
-
11
-
-
84924066675
-
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates
-
11 Meyer, K, Ferraiuolo, L, Schmelzer, L, Braun, L, McGovern, V, Likhite, S, et al. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Mol Ther 23 (2015), 477–487.
-
(2015)
Mol Ther
, vol.23
, pp. 477-487
-
-
Meyer, K.1
Ferraiuolo, L.2
Schmelzer, L.3
Braun, L.4
McGovern, V.5
Likhite, S.6
-
12
-
-
79957882063
-
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
-
12 Fu, H, Dirosario, J, Killedar, S, Zaraspe, K, McCarty, DM, Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol Ther 19 (2011), 1025–1033.
-
(2011)
Mol Ther
, vol.19
, pp. 1025-1033
-
-
Fu, H.1
Dirosario, J.2
Killedar, S.3
Zaraspe, K.4
McCarty, D.M.5
-
13
-
-
79955574241
-
Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder
-
13 Spampanato, C, De Leonibus, E, Dama, P, Gargiulo, A, Fraldi, A, Sorrentino, NC, et al. Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder. Mol Ther 19 (2011), 860–869.
-
(2011)
Mol Ther
, vol.19
, pp. 860-869
-
-
Spampanato, C.1
De Leonibus, E.2
Dama, P.3
Gargiulo, A.4
Fraldi, A.5
Sorrentino, N.C.6
-
14
-
-
84872077107
-
Intravenous delivery of adeno-associated viral vector serotype 9 mediates effective gene expression in ischemic stroke lesion and brain angiogenic foci
-
14 Shen, F, Kuo, R, Milon-Camus, M, Han, Z, Jiang, L, Young, WL, et al. Intravenous delivery of adeno-associated viral vector serotype 9 mediates effective gene expression in ischemic stroke lesion and brain angiogenic foci. Stroke 44 (2013), 252–254.
-
(2013)
Stroke
, vol.44
, pp. 252-254
-
-
Shen, F.1
Kuo, R.2
Milon-Camus, M.3
Han, Z.4
Jiang, L.5
Young, W.L.6
-
15
-
-
85176871116
-
-
15 Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 < https://clinicaltrials.gov/ct2/show/NCT02122952>.
-
-
-
-
16
-
-
78650903887
-
Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed
-
16 Wang, DB, Dayton, RD, Henning, PP, Cain, CD, Zhao, LR, Schrott, LM, et al. Expansive gene transfer in the rat CNS rapidly produces amyotrophic lateral sclerosis relevant sequelae when TDP-43 is overexpressed. Mol Ther 18 (2010), 2064–2074.
-
(2010)
Mol Ther
, vol.18
, pp. 2064-2074
-
-
Wang, D.B.1
Dayton, R.D.2
Henning, P.P.3
Cain, C.D.4
Zhao, L.R.5
Schrott, L.M.6
-
17
-
-
84879687753
-
Selective forelimb impairment in rats expressing a pathological TDP-43 25 kDa C-terminal fragment to mimic amyotrophic lateral sclerosis
-
17 Dayton, RD, Gitcho, MA, Orchard, EA, Wilson, JD, Wang, DB, Cain, CD, et al. Selective forelimb impairment in rats expressing a pathological TDP-43 25 kDa C-terminal fragment to mimic amyotrophic lateral sclerosis. Mol Ther 21 (2013), 1324–1334.
-
(2013)
Mol Ther
, vol.21
, pp. 1324-1334
-
-
Dayton, R.D.1
Gitcho, M.A.2
Orchard, E.A.3
Wilson, J.D.4
Wang, D.B.5
Cain, C.D.6
-
18
-
-
84920541142
-
Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis
-
18 Jackson, KL, Dayton, RD, Orchard, EA, Ju, S, Ringe, D, Petsko, GA, et al. Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene Ther 22 (2015), 20–28.
-
(2015)
Gene Ther
, vol.22
, pp. 20-28
-
-
Jackson, K.L.1
Dayton, R.D.2
Orchard, E.A.3
Ju, S.4
Ringe, D.5
Petsko, G.A.6
-
19
-
-
84903760379
-
Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10
-
19 Yang, B, Li, S, Wang, H, Guo, Y, Gessler, DJ, Cao, C, et al. Global CNS transduction of adult mice by intravenously delivered rAAVrh.8 and rAAVrh.10 and nonhuman primates by rAAVrh.10. Mol Ther 22 (2014), 1299–1309.
-
(2014)
Mol Ther
, vol.22
, pp. 1299-1309
-
-
Yang, B.1
Li, S.2
Wang, H.3
Guo, Y.4
Gessler, D.J.5
Cao, C.6
-
20
-
-
84881293648
-
Mouse gender influences brain transduction by intravascularly administered AAV9
-
20 Maguire, CA, Crommentuijn, MH, Mu, D, Hudry, E, Serrano-Pozo, A, Hyman, BT, et al. Mouse gender influences brain transduction by intravascularly administered AAV9. Mol Ther 21 (2013), 1470–1471.
-
(2013)
Mol Ther
, vol.21
, pp. 1470-1471
-
-
Maguire, C.A.1
Crommentuijn, M.H.2
Mu, D.3
Hudry, E.4
Serrano-Pozo, A.5
Hyman, B.T.6
-
21
-
-
0037818636
-
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway
-
21 Davidoff, AM, Ng, CY, Zhou, J, Spence, Y, Nathwani, AC, Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. Blood 102 (2003), 480–488.
-
(2003)
Blood
, vol.102
, pp. 480-488
-
-
Davidoff, A.M.1
Ng, C.Y.2
Zhou, J.3
Spence, Y.4
Nathwani, A.C.5
-
22
-
-
33745667822
-
Sex and estrous cycle stage influence the efficiency of AAV-mediated gene transfer in the rodent brain. Abstract
-
22 Dodge, JC, Clarke, J, Passini, MA, Song, A, O'Riordan, CR, Cheng, SH, et al. Sex and estrous cycle stage influence the efficiency of AAV-mediated gene transfer in the rodent brain. Abstract. Mol Ther 11 (2005), S192–S193.
-
(2005)
Mol Ther
, vol.11
, pp. S192-S193
-
-
Dodge, J.C.1
Clarke, J.2
Passini, M.A.3
Song, A.4
O'Riordan, C.R.5
Cheng, S.H.6
-
23
-
-
33749632259
-
Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis
-
23 Neumann, M, Sampathu, DM, Kwong, LK, Truax, AC, Micsenyi, MC, Chou, TT, et al. Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Science 314 (2006), 130–133.
-
(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
-
24
-
-
84864877570
-
Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs
-
24 Federici, T, Taub, JS, Baum, GR, Gray, SJ, Grieger, JC, Matthews, KA, et al. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther 19 (2012), 852–859.
-
(2012)
Gene Ther
, vol.19
, pp. 852-859
-
-
Federici, T.1
Taub, J.S.2
Baum, G.R.3
Gray, S.J.4
Grieger, J.C.5
Matthews, K.A.6
-
25
-
-
84860171925
-
Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates
-
25 Samaranch, L, Salegio, EA, San Sebastian, W, Kells, AP, Foust, KD, Bringas, JR, et al. Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Hum Gene Ther 23 (2012), 382–389.
-
(2012)
Hum Gene Ther
, vol.23
, pp. 382-389
-
-
Samaranch, L.1
Salegio, E.A.2
San Sebastian, W.3
Kells, A.P.4
Foust, K.D.5
Bringas, J.R.6
-
26
-
-
84904412918
-
Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy
-
26 Passini, MA, Bu, J, Richards, AM, Treleaven, CM, Sullivan, JA, O'Riordan, CR, et al. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy. Hum Gene Ther 25 (2014), 619–630.
-
(2014)
Hum Gene Ther
, vol.25
, pp. 619-630
-
-
Passini, M.A.1
Bu, J.2
Richards, A.M.3
Treleaven, C.M.4
Sullivan, J.A.5
O'Riordan, C.R.6
-
27
-
-
85015257444
-
A single injection of recombinant adeno-associated virus into the lumbar cistern delivers transgene expression throughout the whole spinal cord
-
(epub ahead of print)
-
27 Guo, Y, Wang, D, Qiao, T, Yang, C, Su, Q, Gao, G, et al. A single injection of recombinant adeno-associated virus into the lumbar cistern delivers transgene expression throughout the whole spinal cord. Mol Neurobiol, 2015 (epub ahead of print).
-
(2015)
Mol Neurobiol
-
-
Guo, Y.1
Wang, D.2
Qiao, T.3
Yang, C.4
Su, Q.5
Gao, G.6
-
28
-
-
0036315645
-
Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain
-
28 Klein, RL, Hamby, ME, Gong, Y, Hirko, AC, Wang, S, Hughes, JA, et al. Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain. Exp Neurol 176 (2002), 66–74.
-
(2002)
Exp Neurol
, vol.176
, pp. 66-74
-
-
Klein, R.L.1
Hamby, M.E.2
Gong, Y.3
Hirko, A.C.4
Wang, S.5
Hughes, J.A.6
-
29
-
-
37549061385
-
AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method
-
29 Klein, RL, Dayton, RD, Tatom, JB, Henderson, KM, Henning, PP, AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method. Mol Ther 16 (2008), 89–96.
-
(2008)
Mol Ther
, vol.16
, pp. 89-96
-
-
Klein, R.L.1
Dayton, R.D.2
Tatom, J.B.3
Henderson, K.M.4
Henning, P.P.5
|