-
1
-
-
77956928344
-
Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia
-
Cavazzana-Calvo M., Payen E., Negre O., Wang G., Hehir K., Fusil F., et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010, 467:318-322.
-
(2010)
Nature
, vol.467
, pp. 318-322
-
-
Cavazzana-Calvo, M.1
Payen, E.2
Negre, O.3
Wang, G.4
Hehir, K.5
Fusil, F.6
-
2
-
-
44049094771
-
Clinical gene therapy using recombinant adeno-associated virus vectors
-
Mueller C., Flotte T.R. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 2008, 15:858-863.
-
(2008)
Gene Ther
, vol.15
, pp. 858-863
-
-
Mueller, C.1
Flotte, T.R.2
-
3
-
-
79954622209
-
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
-
Mingozzi F., High K.A. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat Rev Genet 2011, 12:341-355.
-
(2011)
Nat Rev Genet
, vol.12
, pp. 341-355
-
-
Mingozzi, F.1
High, K.A.2
-
4
-
-
0040417435
-
Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood
-
Zhou S.Z., Cooper S., Kang L.Y., Ruggieri L., Heimfeld S., Srivastava A., et al. Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. JExp Med 1994, 179:1867-1875.
-
(1994)
JExp Med
, vol.179
, pp. 1867-1875
-
-
Zhou, S.Z.1
Cooper, S.2
Kang, L.Y.3
Ruggieri, L.4
Heimfeld, S.5
Srivastava, A.6
-
5
-
-
0027992020
-
Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells
-
Goodman S., Xiao X., Donahue R.E., Moulton A., Miller J., Walsh C., et al. Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 1994, 84:1492-1500.
-
(1994)
Blood
, vol.84
, pp. 1492-1500
-
-
Goodman, S.1
Xiao, X.2
Donahue, R.E.3
Moulton, A.4
Miller, J.5
Walsh, C.6
-
6
-
-
0029945425
-
Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction
-
Fisher-Adams G., Wong K.K., Podsakoff G., Forman S.J., Chatterjee S. Integration of adeno-associated virus vectors in CD34+ human hematopoietic progenitor cells after transduction. Blood 1996, 88:492-504.
-
(1996)
Blood
, vol.88
, pp. 492-504
-
-
Fisher-Adams, G.1
Wong, K.K.2
Podsakoff, G.3
Forman, S.J.4
Chatterjee, S.5
-
7
-
-
0033559749
-
Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors
-
Chatterjee S., Li W., Wong C.A., Fisher-Adams G., Lu D., Guha M., et al. Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors. Blood 1999, 93:1882-1894.
-
(1999)
Blood
, vol.93
, pp. 1882-1894
-
-
Chatterjee, S.1
Li, W.2
Wong, C.A.3
Fisher-Adams, G.4
Lu, D.5
Guha, M.6
-
8
-
-
80955180060
-
Recombinant AAV2-mediated beta-globin expression in human fetal hematopoietic cells from the aborted fetuses with beta-thalassemia major
-
Tian J., Wang F., Xue J.F., Zhao F., Song L.J., Tan M.Q. Recombinant AAV2-mediated beta-globin expression in human fetal hematopoietic cells from the aborted fetuses with beta-thalassemia major. Int J Hematol 2011, 93:691-699.
-
(2011)
Int J Hematol
, vol.93
, pp. 691-699
-
-
Tian, J.1
Wang, F.2
Xue, J.F.3
Zhao, F.4
Song, L.J.5
Tan, M.Q.6
-
9
-
-
34547508637
-
Quiescent subpopulations of human CD34-positive hematopoietic stem cells are preferred targets for stable recombinant adeno-associated virus type 2 transduction
-
Paz H., Wong C.A., Li W., Santat L., Wong K.K., Chatterjee S. Quiescent subpopulations of human CD34-positive hematopoietic stem cells are preferred targets for stable recombinant adeno-associated virus type 2 transduction. Hum Gene Ther 2007, 18:614-626.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 614-626
-
-
Paz, H.1
Wong, C.A.2
Li, W.3
Santat, L.4
Wong, K.K.5
Chatterjee, S.6
-
10
-
-
0033995253
-
Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV
-
Nathwani A.C., Hanawa H., Vandergriff J., Kelly P., Vanin E.F., Nienhuis A.W. Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV. Gene Ther 2000, 7:183-195.
-
(2000)
Gene Ther
, vol.7
, pp. 183-195
-
-
Nathwani, A.C.1
Hanawa, H.2
Vandergriff, J.3
Kelly, P.4
Vanin, E.F.5
Nienhuis, A.W.6
-
11
-
-
0031058475
-
Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line
-
Malik P., McQuiston S.A., Yu X.J., Pepper K.A., Krall W.J., Podsakoff G.M., et al. Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. JVirol 1997, 71:1776-1783.
-
(1997)
JVirol
, vol.71
, pp. 1776-1783
-
-
Malik, P.1
McQuiston, S.A.2
Yu, X.J.3
Pepper, K.A.4
Krall, W.J.5
Podsakoff, G.M.6
-
12
-
-
0030666460
-
Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results
-
Alexander I.E., Russell D.W., Miller A.D. Transfer of contaminants in adeno-associated virus vector stocks can mimic transduction and lead to artifactual results. Hum Gene Ther 1997, 8:1911-1920.
-
(1997)
Hum Gene Ther
, vol.8
, pp. 1911-1920
-
-
Alexander, I.E.1
Russell, D.W.2
Miller, A.D.3
-
13
-
-
0036253749
-
Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors
-
Srivastava A. Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors. JCell Biochem 2002, 38:39-45.
-
(2002)
JCell Biochem
, vol.38
, pp. 39-45
-
-
Srivastava, A.1
-
14
-
-
22144476522
-
Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions
-
Srivastava A. Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: problems and solutions. Hum Gene Ther 2005, 16:792-798.
-
(2005)
Hum Gene Ther
, vol.16
, pp. 792-798
-
-
Srivastava, A.1
-
15
-
-
0033885967
-
Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors
-
Handa A., Muramatsu S., Qiu J., Mizukami H., Brown K.E. Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors. JGen Virol 2000, 81(Pt 8):2077-2084.
-
(2000)
JGen Virol
, vol.81
, pp. 2077-2084
-
-
Handa, A.1
Muramatsu, S.2
Qiu, J.3
Mizukami, H.4
Brown, K.E.5
-
16
-
-
77955502649
-
Gene transfer intohuman cord blood-derived CD34(+) cells by adeno-associated viral vectors
-
Schuhmann N.K., Pozzoli O., Sallach J., Huber A., Avitabile D., Perabo L., et al. Gene transfer intohuman cord blood-derived CD34(+) cells by adeno-associated viral vectors. Exp Hematol 2010, 38:707-717.
-
(2010)
Exp Hematol
, vol.38
, pp. 707-717
-
-
Schuhmann, N.K.1
Pozzoli, O.2
Sallach, J.3
Huber, A.4
Avitabile, D.5
Perabo, L.6
-
17
-
-
75449109000
-
Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34(+) peripheral blood progenitor cells
-
Veldwijk M.R., Sellner L., Stiefelhagen M., Kleinschmidt J.A., Laufs S., Topaly J., et al. Pseudotyped recombinant adeno-associated viral vectors mediate efficient gene transfer into primary human CD34(+) peripheral blood progenitor cells. Cytotherapy 2010, 12:107-112.
-
(2010)
Cytotherapy
, vol.12
, pp. 107-112
-
-
Veldwijk, M.R.1
Sellner, L.2
Stiefelhagen, M.3
Kleinschmidt, J.A.4
Laufs, S.5
Topaly, J.6
-
18
-
-
33645452860
-
Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5
-
Zhong L., Li W., Li Y., Zhao W., Wu J., Li B., et al. Evaluation of primitive murine hematopoietic stem and progenitor cell transduction in vitro and in vivo by recombinant adeno-associated virus vector serotypes 1 through 5. Hum Gene Ther 2006, 17:321-333.
-
(2006)
Hum Gene Ther
, vol.17
, pp. 321-333
-
-
Zhong, L.1
Li, W.2
Li, Y.3
Zhao, W.4
Wu, J.5
Li, B.6
-
19
-
-
41149124496
-
Recombinant self-complementary adeno-associated virus serotype vector-mediated hematopoietic stem cell transduction and lineage-restricted, long-term transgene expression in a murine serial bone marrow transplantation model
-
Maina N., Han Z., Li X., Hu Z., Zhong L., Bischof D., et al. Recombinant self-complementary adeno-associated virus serotype vector-mediated hematopoietic stem cell transduction and lineage-restricted, long-term transgene expression in a murine serial bone marrow transplantation model. Hum Gene Ther 2008, 19:376-383.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 376-383
-
-
Maina, N.1
Han, Z.2
Li, X.3
Hu, Z.4
Zhong, L.5
Bischof, D.6
-
20
-
-
45549090635
-
Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses
-
Zhong L., Li B., Mah C.S., Govindasamy L., Agbandje-McKenna M., Cooper M., et al. Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses. Proc Natl Acad Sci U S A 2008, 105:7827-7832.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 7827-7832
-
-
Zhong, L.1
Li, B.2
Mah, C.S.3
Govindasamy, L.4
Agbandje-McKenna, M.5
Cooper, M.6
-
21
-
-
77957838569
-
Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2
-
Kauss M.A., Smith L.J., Zhong L., Srivastava A., Wong K.K., Chatterjee S. Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2. Hum Gene Ther 2010, 21:1129-1136.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1129-1136
-
-
Kauss, M.A.1
Smith, L.J.2
Zhong, L.3
Srivastava, A.4
Wong, K.K.5
Chatterjee, S.6
-
22
-
-
84859610928
-
Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells
-
Cheng B., Ling C., Dai Y., Lu Y., Glushakova L.G., Gee S.W., et al. Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells. Gene Ther 2012, 19:375-384.
-
(2012)
Gene Ther
, vol.19
, pp. 375-384
-
-
Cheng, B.1
Ling, C.2
Dai, Y.3
Lu, Y.4
Glushakova, L.G.5
Gee, S.W.6
-
23
-
-
77958102381
-
Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle
-
Qiao C., Zhang W., Yuan Z., Shin J.H., Li J., Jayandharan G.R., et al. Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle. Hum Gene Ther 2010, 21:1343-1348.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1343-1348
-
-
Qiao, C.1
Zhang, W.2
Yuan, Z.3
Shin, J.H.4
Li, J.5
Jayandharan, G.R.6
-
24
-
-
78650082356
-
Optimized transduction of human monocyte-derived dendritic cells by recombinant adeno-associated virus serotype 6
-
Ussher J.E., Taylor J.A. Optimized transduction of human monocyte-derived dendritic cells by recombinant adeno-associated virus serotype 6. Hum Gene Ther 2010, 21:1675-1686.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1675-1686
-
-
Ussher, J.E.1
Taylor, J.A.2
-
25
-
-
84856975718
-
Enhanced gene delivery tothe neonatal retina through systemic administration of tyrosine-mutated AAV9
-
Dalkara D., Byrne L.C., Lee T., Hoffmann N.V., Schaffer D.V., Flannery JG. Enhanced gene delivery tothe neonatal retina through systemic administration of tyrosine-mutated AAV9. Gene Ther 2012, 19:176-181.
-
(2012)
Gene Ther
, vol.19
, pp. 176-181
-
-
Dalkara, D.1
Byrne, L.C.2
Lee, T.3
Hoffmann, N.V.4
Schaffer, D.V.5
Flannery, J.G.6
-
26
-
-
2642642141
-
Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
-
Xiao X., Li J., Samulski R.J. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. JVirol 1998, 72:2224-2232.
-
(1998)
JVirol
, vol.72
, pp. 2224-2232
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
27
-
-
23244466031
-
Effect of age on the frequency, cell cycle, and lineage maturation of rhesus monkey (Macaca mulatta) CD34+ and hematopoietic progenitor cells
-
Lee C.C., Fletcher M.D., Tarantal A.F. Effect of age on the frequency, cell cycle, and lineage maturation of rhesus monkey (Macaca mulatta) CD34+ and hematopoietic progenitor cells. Pediatr Res 2005, 58:315-322.
-
(2005)
Pediatr Res
, vol.58
, pp. 315-322
-
-
Lee, C.C.1
Fletcher, M.D.2
Tarantal, A.F.3
-
28
-
-
34548610909
-
Asingle injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease
-
Cearley C.N., Wolfe J.H. Asingle injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. JNeurosci 2007, 27:9928-9940.
-
(2007)
JNeurosci
, vol.27
, pp. 9928-9940
-
-
Cearley, C.N.1
Wolfe, J.H.2
-
29
-
-
23344432113
-
Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice
-
Santat L., Paz H., Wong C., Li L., Macer J., Forman S., et al. Recombinant AAV2 transduction of primitive human hematopoietic stem cells capable of serial engraftment in immune-deficient mice. Proc Natl Acad Sci U S A 2005, 102:11053-11058.
-
(2005)
Proc Natl Acad Sci U S A
, vol.102
, pp. 11053-11058
-
-
Santat, L.1
Paz, H.2
Wong, C.3
Li, L.4
Macer, J.5
Forman, S.6
-
30
-
-
0032506708
-
Long-term genetic modification of rhesus monkey hematopoietic cells following transplantation of adenoassociated virus vector-transduced CD34+ cells
-
Schimmenti S., Boesen J., Claassen E.A., Valerio D., Einerhand M.P. Long-term genetic modification of rhesus monkey hematopoietic cells following transplantation of adenoassociated virus vector-transduced CD34+ cells. Hum Gene Ther 1998, 9:2727-2734.
-
(1998)
Hum Gene Ther
, vol.9
, pp. 2727-2734
-
-
Schimmenti, S.1
Boesen, J.2
Claassen, E.A.3
Valerio, D.4
Einerhand, M.P.5
-
31
-
-
0030879933
-
Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation
-
Ponnazhagan S., Mukherjee P., Wang X.S., Qing K., Kube D.M., Mah C., et al. Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation. JVirol 1997, 71:8262-8267.
-
(1997)
JVirol
, vol.71
, pp. 8262-8267
-
-
Ponnazhagan, S.1
Mukherjee, P.2
Wang, X.S.3
Qing, K.4
Kube, D.M.5
Mah, C.6
-
32
-
-
77953229919
-
Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6
-
Weller M.L., Amornphimoltham P., Schmidt M., Wilson P.A., Gutkind J.S., Chiorini J.A. Epidermal growth factor receptor is a co-receptor for adeno-associated virus serotype 6. Nat Med 2010, 16:662-664.
-
(2010)
Nat Med
, vol.16
, pp. 662-664
-
-
Weller, M.L.1
Amornphimoltham, P.2
Schmidt, M.3
Wilson, P.A.4
Gutkind, J.S.5
Chiorini, J.A.6
-
33
-
-
0025149041
-
Expression of epidermal-growth-factor receptor in the K562 cell line by transfection: altered receptor biochemistry
-
Allen H., Hsuan J., Clark S., Maziarz R., Waterfield M.D., Flavell R.A. Expression of epidermal-growth-factor receptor in the K562 cell line by transfection: altered receptor biochemistry. Biochem J 1990, 271:785-790.
-
(1990)
Biochem J
, vol.271
, pp. 785-790
-
-
Allen, H.1
Hsuan, J.2
Clark, S.3
Maziarz, R.4
Waterfield, M.D.5
Flavell, R.A.6
-
34
-
-
84864008603
-
Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6
-
Denard J., Beley C., Kotin R., Lai-Kuen R., Blot S., Leh H., et al. Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6. JVirol 2012, 86:6620-6631.
-
(2012)
JVirol
, vol.86
, pp. 6620-6631
-
-
Denard, J.1
Beley, C.2
Kotin, R.3
Lai-Kuen, R.4
Blot, S.5
Leh, H.6
-
35
-
-
0036828086
-
Adenovirus-facilitated nuclear translocation of adeno-associated virus type2
-
Xiao W., Warrington K.H., Hearing P., Hearing P., Hughes J., Muzyczka N. Adenovirus-facilitated nuclear translocation of adeno-associated virus type2. JVirol 2002, 76:11505-11517.
-
(2002)
JVirol
, vol.76
, pp. 11505-11517
-
-
Xiao, W.1
Warrington, K.H.2
Hearing, P.3
Hearing, P.4
Hughes, J.5
Muzyczka, N.6
-
36
-
-
84869015419
-
Cytoplasmic trafficking, endosomal escape, and peri-nuclear accumulation of AAV2 particles are facilitated by microtubule network
-
Xiao P.J., Samulski R.J. Cytoplasmic trafficking, endosomal escape, and peri-nuclear accumulation of AAV2 particles are facilitated by microtubule network. JVirol 2012, 86:10462-10473.
-
(2012)
JVirol
, vol.86
, pp. 10462-10473
-
-
Xiao, P.J.1
Samulski, R.J.2
-
37
-
-
78650898983
-
High-efficiency transduction and correction of murine hemophilia Busing AAV2 vectors devoid of multiple surface-exposed tyrosines
-
Markusic D.M., Herzog R.W., Aslanidi G.V., Hoffman B.E., Li B., Li M., et al. High-efficiency transduction and correction of murine hemophilia Busing AAV2 vectors devoid of multiple surface-exposed tyrosines. Mol Ther 2010, 18:2048-2056.
-
(2010)
Mol Ther
, vol.18
, pp. 2048-2056
-
-
Markusic, D.M.1
Herzog, R.W.2
Aslanidi, G.V.3
Hoffman, B.E.4
Li, B.5
Li, M.6
-
38
-
-
84861189907
-
High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors
-
Aslanidi G.V., Rivers A.E., Ortiz L., Govindasamy L., Ling C., Jayandharan G.R., et al. High-efficiency transduction of human monocyte-derived dendritic cells by capsid-modified recombinant AAV2 vectors. Vaccine 2012, 30:3908-3917.
-
(2012)
Vaccine
, vol.30
, pp. 3908-3917
-
-
Aslanidi, G.V.1
Rivers, A.E.2
Ortiz, L.3
Govindasamy, L.4
Ling, C.5
Jayandharan, G.R.6
-
39
-
-
84875092627
-
Optimization of recombinant adeno-associated virus 2 (AAV2) vectors for gene therapy: the final threshold?
-
Aslanidi G.V., Rivers A.E., Ortiz L., Song L., Govindasamy L., Tan M., et al. Optimization of recombinant adeno-associated virus 2 (AAV2) vectors for gene therapy: the final threshold?. PloS One 2013, 8:e59142.
-
(2013)
PloS One
, vol.8
, pp. e59142
-
-
Aslanidi, G.V.1
Rivers, A.E.2
Ortiz, L.3
Song, L.4
Govindasamy, L.5
Tan, M.6
-
40
-
-
84875040149
-
High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo
-
Song L., Li X., Jayandharan G.R., Wang Y., Aslanidi G.V., Ling C., et al. High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo. PloS One 2013, 8:e58757.
-
(2013)
PloS One
, vol.8
, pp. e58757
-
-
Song, L.1
Li, X.2
Jayandharan, G.R.3
Wang, Y.4
Aslanidi, G.V.5
Ling, C.6
|