-
1
-
-
84890120669
-
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in canavan mice
-
Ahmed, S. S., Li, H., Cao, C., Sikoglu, E. M., Denninger, A. R., Su, Q., et al. (2013). A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in canavan mice. Mol. Ther. 21, 2136-2147. doi: 10.1038/mt.2013.138
-
(2013)
Mol. Ther
, vol.21
, pp. 2136-2147
-
-
Ahmed, S.S.1
Li, H.2
Cao, C.3
Sikoglu, E.M.4
Denninger, A.R.5
Su, Q.6
-
3
-
-
84873410199
-
Intramuscular AAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice
-
Benkhelifa-Ziyyat, S., Besse, A., Roda, M., Duqué, S., Astord, S., Carcenac, R., et al. (2013). Intramuscular AAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol. Ther. 21, 282-290. doi: 10.1038/mt.2012.261
-
(2013)
Mol. Ther
, vol.21
, pp. 282-290
-
-
Benkhelifa-Ziyyat, S.1
Besse, A.2
Roda, M.3
Duqué, S.4
Astord, S.5
Carcenac, R.6
-
4
-
-
80455173951
-
Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders
-
Bevan, A. K., Duque, S., Foust, K. D., Morales, P. R., Braun, L., Schmelzer, L., et al. (2011). Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Mol. Ther. 19, 1971-1980. doi: 10.1038/mt.2011.157
-
(2011)
Mol. Ther
, vol.19
, pp. 1971-1980
-
-
Bevan, A.K.1
Duque, S.2
Foust, K.D.3
Morales, P.R.4
Braun, L.5
Schmelzer, L.6
-
5
-
-
84900032549
-
Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats
-
Bucher, T., Dubreil, L., Colle, M. A., Maquigneau, M., Deniaud, J., Ledevin, M., et al. (2014). Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats. Gene Ther. 21, 522-528. doi: 10.1038/gt.2014.16
-
(2014)
Gene Ther
, vol.21
, pp. 522-528
-
-
Bucher, T.1
Dubreil, L.2
Colle, M.A.3
Maquigneau, M.4
Deniaud, J.5
Ledevin, M.6
-
6
-
-
32944459983
-
Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain
-
Cearley, C. N., and Wolfe, J. H. (2006). Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain.Mol. Ther. 13, 528-537. doi: 10.1016/j.ymthe.2005.11.015
-
(2006)
Mol. Ther
, vol.13
, pp. 528-537
-
-
Cearley, C.N.1
Wolfe, J.H.2
-
7
-
-
73449128979
-
Safety and tolerability of putaminal AADC gene therapy for Parkinson disease
-
Christine, C. W., Starr, P. A., Larson, P. S., Eberling, J. L., Jagust, W. J., Hawkins, R. A., et al. (2009). Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology 73, 1662-1669. doi: 10.1212/WNL.0b013e3181c29356
-
(2009)
Neurology
, vol.73
, pp. 1662-1669
-
-
Christine, C.W.1
Starr, P.A.2
Larson, P.S.3
Eberling, J.L.4
Jagust, W.J.5
Hawkins, R.A.6
-
8
-
-
0034724209
-
Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system
-
Davidson, B. L., Stein, C. S., Heth, J. A., Martins, I., Kotin, R. M., Derksen, T. A., et al. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc. Natl. Acad. Sci. U.S.A. 97, 3428-3432. doi: 10.1073/pnas.97.7.3428
-
(2000)
Proc. Natl. Acad. Sci. U.S.A
, vol.97
, pp. 3428-3432
-
-
Davidson, B.L.1
Stein, C.S.2
Heth, J.A.3
Martins, I.4
Kotin, R.M.5
Derksen, T.A.6
-
9
-
-
0011039347
-
Morphological aspects of the barriers
-
(Boca Raton, FL: CRC Press)
-
Davson, H., and Segal, M. B. (1996). "Morphological aspects of the barriers," inPhysiology of the CSF and Blood-brain Barrier (Boca Raton, FL: CRC Press), 93-192.
-
(1996)
Physiology of the CSF and Blood-brain Barrier
, pp. 93-192
-
-
Davson, H.1
Segal, M.B.2
-
10
-
-
78751700314
-
Intravenous AAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice
-
Dominguez, E., Marais, T., Chatauret, N., Benkhelifa-Ziyyat, S., Duque, S., Ravassard, P., et al. (2011). Intravenous AAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum. Mol. Genet. 20, 681-693. doi: 10.1093/hmg/ddq514
-
(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
-
11
-
-
67649861393
-
Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons
-
Duqué, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A. M., et al. (2009). Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther. 17, 1187-1196. doi: 10.1038/mt.2009.71
-
(2009)
Mol. Ther
, vol.17
, pp. 1187-1196
-
-
Duqué, S.1
Joussemet, B.2
Riviere, C.3
Marais, T.4
Dubreil, L.5
Douar, A.M.6
-
12
-
-
0029059327
-
HIV infection of human choroid plexus: A possible mechanism of viral entry into the CNS
-
Falangola, M. F., Hanly, A., Galvao-Castro, B., and Petito, C. K. (1995). HIV infection of human choroid plexus: a possible mechanism of viral entry into the CNS. J. Neuropathol. Exp. Neurol. 54, 497-503. doi: 10.1097/00005072-199507000-00003
-
(1995)
J. Neuropathol. Exp. Neurol
, vol.54
, pp. 497-503
-
-
Falangola, M.F.1
Hanly, A.2
Galvao-Castro, B.3
Petito, C.K.4
-
13
-
-
60149106907
-
Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
-
Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., and Kaspar, B. K. (2009). Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol. 27, 59-65. doi: 10.1038/nbt.1515
-
(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
-
14
-
-
77749249680
-
Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
-
Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., et al. (2010). Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat. Biotechnol. 28, 271-274. doi: 10.1038/nbt.1610
-
(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
-
15
-
-
79957882063
-
Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery
-
Fu, H., Dirosario, J., Killedar, S., Zaraspe, K., and McCarty, D. M. (2011). Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery. Mol. Ther. 19, 1025-1033. doi: 10.1038/mt.2011.34
-
(2011)
Mol. Ther
, vol.19
, pp. 1025-1033
-
-
Fu, H.1
Dirosario, J.2
Killedar, S.3
Zaraspe, K.4
McCarty, D.M.5
-
16
-
-
33748283924
-
An approximately 140-kb deletion associated with feline spinal muscular atrophy implies an essential LIX1 function for motor neuron survival
-
Fyfe, J. C., Menotti-Raymond, M., David, V. A., Brichta, L., Schäffer, A. A., Agarwala, R., et al. (2006). An approximately 140-kb deletion associated with feline spinal muscular atrophy implies an essential LIX1 function for motor neuron survival.Genome Res. 16, 1084-1090. doi: 10.1101/gr.5268806
-
(2006)
Genome Res
, vol.16
, pp. 1084-1090
-
-
Fyfe, J.C.1
Menotti-Raymond, M.2
David, V.A.3
Brichta, L.4
Schäffer, A.A.5
Agarwala, R.6
-
17
-
-
0037947557
-
Adeno-associated viruses undergo substantial evolution in primates during natural infections
-
Gao, G., Alvira, M. R., Somanathan, S., Lu, Y., Vandenberghe, L. H., Rux, J. J., et al. (2003). Adeno-associated viruses undergo substantial evolution in primates during natural infections. Proc. Natl. Acad. Sci. U.S.A. 100, 6081-6086. doi: 10.1073/pnas.0937739100
-
(2003)
Proc. Natl. Acad. Sci. U.S.A
, vol.100
, pp. 6081-6086
-
-
Gao, G.1
Alvira, M.R.2
Somanathan, S.3
Lu, Y.4
Vandenberghe, L.H.5
Rux, J.J.6
-
18
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao, G. P., Alvira, M. R., Wang, L., Calcedo, R., Johnston, J., and Wilson, J. M. (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U.S.A. 99, 11854-11859. doi: 10.1073/pnas.182412299
-
(2002)
Proc. Natl. Acad. Sci. U.S.A
, vol.99
, pp. 11854-11859
-
-
Gao, G.P.1
Alvira, M.R.2
Wang, L.3
Calcedo, R.4
Johnston, J.5
Wilson, J.M.6
-
19
-
-
79957888016
-
Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates
-
Gray, S. J., Matagne, V., Bachaboina, L., Yadav, S., Ojeda, S. R., and Samulski, R. J. (2011). Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates. Mol. Ther. 19, 1058-1069. doi: 10.1038/mt.2011.72
-
(2011)
Mol. Ther
, vol.19
, pp. 1058-1069
-
-
Gray, S.J.1
Matagne, V.2
Bachaboina, L.3
Yadav, S.4
Ojeda, S.R.5
Samulski, R.J.6
-
20
-
-
84857602982
-
Adeno-associated virus vectorology, manufacturing, and clinical applications
-
Grieger, J. C., and Samulski, R. J. (2012). Adeno-associated virus vectorology, manufacturing, and clinical applications. Meth. Enzymol. 507, 229-254. doi: 10.1016/B978-0-12-386509-0.00012-0
-
(2012)
Meth. Enzymol
, vol.507
, pp. 229-254
-
-
Grieger, J.C.1
Samulski, R.J.2
-
21
-
-
84920266970
-
Spectrum of neuropathophysiology in spinal muscular atrophy type I
-
Harding, B. N., Kariya, S., Monani, U. R., Chung, W. K., Benton, M., Yum, S. W., et al. (2015). Spectrum of neuropathophysiology in spinal muscular atrophy type I. J. Neuropathol. Exp. Neurol. 74, 15-24. doi: 10.1097/NEN.0000000000000144
-
(2015)
J. Neuropathol. Exp. Neurol
, vol.74
, pp. 15-24
-
-
Harding, B.N.1
Kariya, S.2
Monani, U.R.3
Chung, W.K.4
Benton, M.5
Yum, S.W.6
-
22
-
-
84926421140
-
Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats
-
Hordeaux, J., Dubreil, L., Deniaud, J., Iacobelli, F., Moreau, S., Ledevin, M., et al. (2015). Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats. Gene Ther. 22, 316-324. doi: 10.1038/gt.2014.121
-
(2015)
Gene Ther
, vol.22
, pp. 316-324
-
-
Hordeaux, J.1
Dubreil, L.2
Deniaud, J.3
Iacobelli, F.4
Moreau, S.5
Ledevin, M.6
-
23
-
-
77956628142
-
RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy
-
Hu, C., Busuttil, R. W., and Lipshutz, G. S. (2010). RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy. J. Gene Med. 12, 766-778. doi: 10.1002/jgm.1496
-
(2010)
J. Gene Med
, vol.12
, pp. 766-778
-
-
Hu, C.1
Busuttil, R.W.2
Lipshutz, G.S.3
-
24
-
-
0027211759
-
Sensory circumventricular organs and brain homeostatic pathways
-
Johnson, A. K., and Gross, P. M. (1993). Sensory circumventricular organs and brain homeostatic pathways. FASEB J. 7, 678-686.
-
(1993)
FASEB J
, vol.7
, pp. 678-686
-
-
Johnson, A.K.1
Gross, P.M.2
-
25
-
-
34250683023
-
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: An open label, phase I trial
-
Kaplitt, M. G., Feigin, A., Tang, C., Fitzsimons, H. L., Mattis, P., Lawlor, P. A., et al. (2007). Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369, 2097-2105. doi: 10.1016/S0140-6736(07)60982-9
-
(2007)
Lancet
, vol.369
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
Fitzsimons, H.L.4
Mattis, P.5
Lawlor, P.A.6
-
26
-
-
37549061385
-
AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: Effects of serotype, promoter and purification method
-
Klein, R. L., Dayton, R. D., Tatom, J. B., Henderson, K. M., and Henning, P. P. (2008). AAV8, 9, Rh10, Rh43 vector gene transfer in the rat brain: effects of serotype, promoter and purification method. Mol. Ther. 16, 89-96. doi: 10.1038/sj.mt.6300331
-
(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
-
27
-
-
20144385587
-
SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN
-
Le, T. T., Pham, L. T., Butchbach, M. E., Zhang, H. L., Monani, U. R., Coovert, D. D., et al. (2005). SMNDelta7, the major product of the centromeric survival motor neuron (SMN2) gene, extends survival in mice with spinal muscular atrophy and associates with full-length SMN. Hum. Mol. Genet. 14, 845-857. doi: 10.1093/hmg/ddi078
-
(2005)
Hum. Mol. Genet
, vol.14
, pp. 845-857
-
-
Le, T.T.1
Pham, L.T.2
Butchbach, M.E.3
Zhang, H.L.4
Monani, U.R.5
Coovert, D.D.6
-
28
-
-
79952740079
-
AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind, sham-surgery controlled, randomised trial
-
LeWitt, P. A., Rezai, A. R., Leehey, M. A., Ojemann, S. G., Flaherty, A. W., Eskandar, E. N., et al. (2011). AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial. Lancet Neurol. 10, 309-319. doi: 10.1016/S1474-4422(11)70039-4
-
(2011)
Lancet Neurol
, vol.10
, pp. 309-319
-
-
LeWitt, P.A.1
Rezai, A.R.2
Leehey, M.A.3
Ojemann, S.G.4
Flaherty, A.W.5
Eskandar, E.N.6
-
29
-
-
6344283047
-
Clinical trials in neurological disorders using AAV vectors: Promises and challenges
-
Mandel, R. J., and Burger, C. (2004). Clinical trials in neurological disorders using AAV vectors: promises and challenges. Curr. Opin. Mol. Ther. 6, 482-490.
-
(2004)
Curr. Opin. Mol. Ther
, vol.6
, pp. 482-490
-
-
Mandel, R.J.1
Burger, C.2
-
30
-
-
41949110690
-
Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: An open-label, phase I trial
-
Marks, W. J. Jr., Ostrem, J. L., Verhagen, L., Starr, P. A., Larson, P. S., Bakay, R. A., et al. (2008). Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial. Lancet Neurol. 7, 400-408. doi: 10.1016/S1474-4422(08)70065-6
-
(2008)
Lancet Neurol
, vol.7
, pp. 400-408
-
-
Marks, W.J.1
Ostrem, J.L.2
Verhagen, L.3
Starr, P.A.4
Larson, P.S.5
Bakay, R.A.6
-
31
-
-
84871994994
-
Systemic delivery of AAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems
-
Mattar, C. N., Waddington, S. N., Biswas, A., Johana, N., Ng, X. W., Fisk, A. S., et al. (2013). Systemic delivery of AAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Gene Ther. 20, 69-83. doi: 10.1038/gt.2011.216
-
(2013)
Gene Ther
, vol.20
, pp. 69-83
-
-
Mattar, C.N.1
Waddington, S.N.2
Biswas, A.3
Johana, N.4
Ng, X.W.5
Fisk, A.S.6
-
32
-
-
0030003480
-
Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
-
McCown, T. J., Xiao, X., Li, J., Breese, G. R., and Samulski, R. J. (1996). Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector. Brain Res. 25, 99-107. doi: 10.1016/0006-8993(95)01488-8
-
(1996)
Brain Res
, vol.25
, pp. 99-107
-
-
McCown, T.J.1
Xiao, X.2
Li, J.3
Breese, G.R.4
Samulski, R.J.5
-
33
-
-
79955467779
-
Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors
-
Miyake, N., Miyake, K., Yamamoto, M., Hirai, Y., and Shimada, T. (2011). Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors. Brain Res. 1389, 19-26. doi: 10.1016/j.brainres.2011.03.014
-
(2011)
Brain Res
, vol.1389
, pp. 19-26
-
-
Miyake, N.1
Miyake, K.2
Yamamoto, M.3
Hirai, Y.4
Shimada, T.5
-
34
-
-
9644265332
-
Two novel adeno-associated viruses from cynomolgus monkey: Pseudotyping characterization of capsid protein
-
Mori, S., Wang, L., Takeuchi, T., and Kanda, T. (2004). Two novel adeno-associated viruses from cynomolgus monkey: pseudotyping characterization of capsid protein.Virology. 330, 375-383. doi: 10.1016/j.virol.2004.10.012
-
(2004)
Virology
, vol.330
, pp. 375-383
-
-
Mori, S.1
Wang, L.2
Takeuchi, T.3
Kanda, T.4
-
35
-
-
33645083312
-
The blood-brain barrier in HIV-associated dementia
-
Pereira, C. F., and Nottet, H. S. L. M. (2000). The blood-brain barrier in HIV-associated dementia. Science 3, 1-8
-
(2000)
Science
, vol.3
, pp. 1-8
-
-
Pereira, C.F.1
Nottet, H.S.L.M.2
-
36
-
-
84884577974
-
Systemic delivery of shRNA by AAV9 provides highly efficient knockdown of ubiquitously expressed GFP in mouse heart, but not liver
-
Piras, B. A., O'Connor, D. M., and French, B. A. (2013). Systemic delivery of shRNA by AAV9 provides highly efficient knockdown of ubiquitously expressed GFP in mouse heart, but not liver. PLoS ONE 8:e75894. doi: 10.1371/journal.pone.0075894
-
(2013)
PLoS ONE
, vol.8
-
-
Piras, B.A.1
O'Connor, D.M.2
French, B.A.3
-
37
-
-
84869093015
-
Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease
-
Rafi, M. A., Rao, H. Z., Luzi, P., Curtis, M. T., and Wenger, D. A. (2012). Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease. Mol. Ther. 20, 2031-2042. doi: 10.1038/mt.2012.153
-
(2012)
Mol. Ther
, vol.20
, pp. 2031-2042
-
-
Rafi, M.A.1
Rao, H.Z.2
Luzi, P.3
Curtis, M.T.4
Wenger, D.A.5
-
38
-
-
84871243601
-
Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer
-
Ruzo, A., Marcó, S., García, M., Villacampa, P., Ribera, A., Ayuso, E., et al. (2012). Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer.Hum. Gene Ther. 23, 1237-1246. doi: 10.1089/hum.2012.029
-
(2012)
Hum. Gene Ther
, vol.23
, pp. 1237-1246
-
-
Ruzo, A.1
Marcó, S.2
García, M.3
Villacampa, P.4
Ribera, A.5
Ayuso, E.6
-
39
-
-
33847211798
-
Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector
-
Sondhi, D., Hackett, N. R., Peterson, D. A., Stratton, J., Baad, M., Travis, K. M., et al. (2007). Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. Mol. Ther. 15, 481-491. doi: 10.1038/sj.mt.6300049
-
(2007)
Mol. Ther
, vol.15
, pp. 481-491
-
-
Sondhi, D.1
Hackett, N.R.2
Peterson, D.A.3
Stratton, J.4
Baad, M.5
Travis, K.M.6
-
40
-
-
84902952909
-
Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: Results of a phase I/II trial
-
Tardieu, M., Zérah, M., Husson, B., De Bournonville, S., Deiva, K., Adamsbaum, C., et al. (2014). Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial. Hum. Gene Ther. 5, 506-516 doi: 10.1089/hum.2013.238
-
(2014)
Hum. Gene Ther
, vol.5
, pp. 506-516
-
-
Tardieu, M.1
Zérah, M.2
Husson, B.3
De Bournonville, S.4
Deiva, K.5
Adamsbaum, C.6
-
41
-
-
84922399417
-
AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy
-
Thwaite, R., Pagès, G., Chillón, M., and Bosch, A. (2015). AAVrh.10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy.Gene Ther. 22, 196-201. doi: 10.1038/gt.2014.103
-
(2015)
Gene Ther
, vol.22
, pp. 196-201
-
-
Thwaite, R.1
Pagès, G.2
Chillón, M.3
Bosch, A.4
-
42
-
-
77955602597
-
Systemic delivery of AAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy
-
Valori, C. F., Ning, K., Wyles, M., Mead, R. J., Grierson, A. J., Shaw, P. J., et al. (2010). Systemic delivery of AAV9 expressing SMN prolongs survival in a model of spinal muscular atrophy. Sci. Transl. Med. 2, 35-42. doi: 10.1126/scitranslmed.3000830
-
(2010)
Sci. Transl. Med
, vol.2
, pp. 35-42
-
-
Valori, C.F.1
Ning, K.2
Wyles, M.3
Mead, R.J.4
Grierson, A.J.5
Shaw, P.J.6
-
43
-
-
84875240244
-
Adeno-associated virus (AAV) gene therapy for neurological disease
-
Weinberg, M. S., Samulski, R. J., and McCown, T. J. (2013). Adeno-associated virus (AAV) gene therapy for neurological disease. Neuropharmacology 69, 82-88. doi: 10.1016/j.neuropharm.2012.03.004
-
(2013)
Neuropharmacology
, vol.69
, pp. 82-88
-
-
Weinberg, M.S.1
Samulski, R.J.2
McCown, T.J.3
-
44
-
-
79954902379
-
Evaluation and optimization of the administration of a selectively replicating herpes simplex viral vector to the brain by convection-enhanced delivery
-
White, E., Bienemann, A., Megraw, L., Bunnun, C., and Gill, S. (2011). Evaluation and optimization of the administration of a selectively replicating herpes simplex viral vector to the brain by convection-enhanced delivery. Cancer Gene Ther. 18, 358-369. doi: 10.1038/cgt.2011.2
-
(2011)
Cancer Gene Ther
, vol.18
, pp. 358-369
-
-
White, E.1
Bienemann, A.2
Megraw, L.3
Bunnun, C.4
Gill, S.5
-
45
-
-
0031779697
-
Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets
-
Wu, P., Phillips, M. I., Bui, J., and Terwilliger, E. F. (1998). Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cell targets.J. Virol. 72, 5919-5926.
-
(1998)
J. Virol
, vol.72
, pp. 5919-5926
-
-
Wu, P.1
Phillips, M.I.2
Bui, J.3
Terwilliger, E.F.4
-
46
-
-
84887084505
-
Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons
-
Yamashita, T., Chai, H. L., Teramoto, S., Tsuji, S., Shimazaki, K., Muramatsu, S., et al. (2013). Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons. EMBO Mol. Med. 5, 1710-1719. doi: 10.1002/emmm.201302935
-
(2013)
EMBO Mol. Med
, vol.5
, pp. 1710-1719
-
-
Yamashita, T.1
Chai, H.L.2
Teramoto, S.3
Tsuji, S.4
Shimazaki, K.5
Muramatsu, S.6
-
47
-
-
79961032418
-
Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system
-
Zhang, H., Yang, B., Mu, X., Ahmed, S. S., Su, Q., He, R., et al. (2011). Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol. Ther. 19, 1440-1448. doi: 10.1038/mt.2011.98
-
(2011)
Mol. Ther
, vol.19
, pp. 1440-1448
-
-
Zhang, H.1
Yang, B.2
Mu, X.3
Ahmed, S.S.4
Su, Q.5
He, R.6
|