-
1
-
-
0037189401
-
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning
-
Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002; 296: 2410-2413.
-
(2002)
Science
, vol.296
, pp. 2410-2413
-
-
Aiuti, A.1
Slavin, S.2
Aker, M.3
Ficara, F.4
Deola, S.5
Mortellaro, A.6
-
2
-
-
78149482538
-
Stem-cell gene therapy for the Wiskott-Aldrich syndrome
-
Boztug K, Schmidt M, Schwarzer A, Banerjee PP, Diez IA, Dewey RA et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N Engl J Med 2010; 363: 1918-1927.
-
(2010)
N Engl J Med
, vol.363
, pp. 1918-1927
-
-
Boztug, K.1
Schmidt, M.2
Schwarzer, A.3
Banerjee, P.P.4
Diez, I.A.5
Dewey, R.A.6
-
4
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006; 12: 401-409.
-
(2006)
Nat Med
, vol.12
, pp. 401-409
-
-
Ott, M.G.1
Schmidt, M.2
Schwarzwaelder, K.3
Stein, S.4
Siler, U.5
Koehl, U.6
-
5
-
-
79956354395
-
Correction of murine {beta}-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion
-
Negre O, Fusil F, Colomb C, Roth S, Gillet-Legrand B, Henri A et al. Correction of murine {beta}-thalassemia after minimal lentiviral gene transfer and homeostatic in vivo erythroid expansion. Blood 2011; 117: 5321-5331.
-
(2011)
Blood
, vol.117
, pp. 5321-5331
-
-
Negre, O.1
Fusil, F.2
Colomb, C.3
Roth, S.4
Gillet-Legrand, B.5
Henri, A.6
-
6
-
-
69849094491
-
A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: Critical determinants for successful correction
-
Perumbeti A, Higashimoto T, Urbinati F, Franco R, Meiselman HJ, Witte D et al. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction. Blood 2009; 114: 1174-1185.
-
(2009)
Blood
, vol.114
, pp. 1174-1185
-
-
Perumbeti, A.1
Higashimoto, T.2
Urbinati, F.3
Franco, R.4
Meiselman, H.J.5
Witte, D.6
-
7
-
-
67649432567
-
Pathogenesis of leukodystrophy for Krabbe disease: Molecular mechanism and clinical treatment
-
Sakai N. Pathogenesis of leukodystrophy for Krabbe disease: molecular mechanism and clinical treatment. Brain Dev 2009; 31: 485-487.
-
(2009)
Brain Dev
, vol.31
, pp. 485-487
-
-
Sakai, N.1
-
8
-
-
22644442838
-
Regulatable gene expression systems for gene therapy applications: Progress and future challenges
-
Goverdhana S, Puntel M, Xiong W, Zirger JM, Barcia C, Curtin JF et al. Regulatable gene expression systems for gene therapy applications: progress and future challenges. Mol Ther 2005; 12: 189-211.
-
(2005)
Mol Ther
, vol.12
, pp. 189-211
-
-
Goverdhana, S.1
Puntel, M.2
Xiong, W.3
Zirger, J.M.4
Barcia, C.5
Curtin, J.F.6
-
9
-
-
79952678001
-
Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemia
-
Heckl D, Wicke DC, Brugman MH, Meyer J, Schambach A, Busche G et al. Lentiviral gene transfer regenerates hematopoietic stem cells in a mouse model for Mpl-deficient aplastic anemia. Blood 2011; 117: 3737-3747.
-
(2011)
Blood
, vol.117
, pp. 3737-3747
-
-
Heckl, D.1
Wicke, D.C.2
Brugman, M.H.3
Meyer, J.4
Schambach, A.5
Busche, G.6
-
10
-
-
36849060584
-
Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state
-
Brown BD, Gentner B, Cantore A, Colleoni S, Amendola M, Zingale A et al. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat Biotechnol 2007; 25: 1457-1467.
-
(2007)
Nat Biotechnol
, vol.25
, pp. 1457-1467
-
-
Brown, B.D.1
Gentner, B.2
Cantore, A.3
Colleoni, S.4
Amendola, M.5
Zingale, A.6
-
11
-
-
67651087321
-
Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications
-
Brown BD, Naldini L. Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications. Nat Rev Genet 2009; 10: 578-585.
-
(2009)
Nat Rev Genet
, vol.10
, pp. 578-585
-
-
Brown, B.D.1
Naldini, L.2
-
12
-
-
73249118950
-
Absolute quantification of microRNAs by using a universal reference
-
Bissels U, Wild S, Tomiuk S, Holste A, Hafner M, Tuschl T et al. Absolute quantification of microRNAs by using a universal reference. RNA 2009; 15: 2375-2384.
-
(2009)
RNA
, vol.15
, pp. 2375-2384
-
-
Bissels, U.1
Wild, S.2
Tomiuk, S.3
Holste, A.4
Hafner, M.5
Tuschl, T.6
-
13
-
-
77957252821
-
Comprehensive microRNA expression profiling of the hematopoietic hierarchy
-
Petriv OI, Kuchenbauer F, Delaney AD, Lecault V, White A, Kent D et al. Comprehensive microRNA expression profiling of the hematopoietic hierarchy. Proc Natl Acad Sci USA 2010; 107: 15443-15448.
-
(2010)
Proc Natl Acad Sci USA
, vol.107
, pp. 15443-15448
-
-
Petriv, O.I.1
Kuchenbauer, F.2
Delaney, A.D.3
Lecault, V.4
White, A.5
Kent, D.6
-
14
-
-
33646564701
-
Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer
-
Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L. Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med 2006; 12: 585-591.
-
(2006)
Nat Med
, vol.12
, pp. 585-591
-
-
Brown, B.D.1
Venneri, M.A.2
Zingale, A.3
Sergi Sergi, L.4
Naldini, L.5
-
15
-
-
36849053006
-
A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice
-
Brown BD, Cantore A, Annoni A, Sergi LS, Lombardo A, Della Valle P et al. A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 2007; 110: 4144-4152.
-
(2007)
Blood
, vol.110
, pp. 4144-4152
-
-
Brown, B.D.1
Cantore, A.2
Annoni, A.3
Sergi, L.S.4
Lombardo, A.5
Della Valle, P.6
-
16
-
-
78449270317
-
Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy
-
Gentner B, Visigalli I, Hiramatsu H, Lechman E, Ungari S, Giustacchini A et al. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci Transl Med 2010; 2: 58ra84.
-
(2010)
Sci Transl Med
, vol.2
-
-
Gentner, B.1
Visigalli, I.2
Hiramatsu, H.3
Lechman, E.4
Ungari, S.5
Giustacchini, A.6
-
17
-
-
33751184381
-
Gene transfer of cytidine deaminase protects myelopoiesis from cytidine analogs in an in vivo murine transplant model
-
Rattmann I, Kleff V, Sorg UR, Bardenheuer W, Brueckner A, Hilger RA et al. Gene transfer of cytidine deaminase protects myelopoiesis from cytidine analogs in an in vivo murine transplant model. Blood 2006; 108: 2965-2971.
-
(2006)
Blood
, vol.108
, pp. 2965-2971
-
-
Rattmann, I.1
Kleff, V.2
Sorg, U.R.3
Bardenheuer, W.4
Brueckner, A.5
Hilger, R.A.6
-
18
-
-
24644489402
-
MicroRNA profiling of the murine hematopoietic system
-
Monticelli S, Ansel KM, Xiao C, Socci ND, Krichevsky AM, Thai TH et al. MicroRNA profiling of the murine hematopoietic system. Genome Biol 2005; 6: R71.
-
(2005)
Genome Biol
, vol.6
-
-
Monticelli, S.1
Ansel, K.M.2
Xiao, C.3
Socci, N.D.4
Krichevsky, A.M.5
Thai, T.H.6
-
19
-
-
79956089851
-
Ordered progression of stage specific miRNA profiles in the mouse B2 B cell lineage
-
Spierings DC, McGoldrick D, Hamilton-Easton AM, Neale G, Murchison EP, Hannon GJ et al. Ordered progression of stage specific miRNA profiles in the mouse B2 B cell lineage. Blood 2011; 117: 5340-5349.
-
(2011)
Blood
, vol.117
, pp. 5340-5349
-
-
Spierings, D.C.1
McGoldrick, D.2
Hamilton-Easton, A.M.3
Neale, G.4
Murchison, E.P.5
Hannon, G.J.6
-
20
-
-
34547941361
-
MiR-150 controls B cell differentiation by targeting the transcription factor c-Myb
-
Xiao C, Calado DP, Galler G, Thai TH, Patterson HC, Wang J et al. MiR-150 controls B cell differentiation by targeting the transcription factor c-Myb. Cell 2007; 131: 146-159.
-
(2007)
Cell
, vol.131
, pp. 146-159
-
-
Xiao, C.1
Calado, D.P.2
Galler, G.3
Thai, T.H.4
Patterson, H.C.5
Wang, J.6
-
21
-
-
33947104955
-
Dynamic regulation of miRNA expression in ordered stages of cellular development
-
Neilson JR, Zheng GX, Burge CB, Sharp PA. Dynamic regulation of miRNA expression in ordered stages of cellular development. Genes Dev 2007; 21: 578-589.
-
(2007)
Genes Dev
, vol.21
, pp. 578-589
-
-
Neilson, J.R.1
Zheng, G.X.2
Burge, C.B.3
Sharp, P.A.4
-
22
-
-
0034669932
-
High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors
-
Salmon P, Kindler V, Ducrey O, Chapuis B, Zubler RH, Trono D. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 2000; 96: 3392-3398.
-
(2000)
Blood
, vol.96
, pp. 3392-3398
-
-
Salmon, P.1
Kindler, V.2
Ducrey, O.3
Chapuis, B.4
Zubler, R.H.5
Trono, D.6
-
23
-
-
0346727524
-
MicroRNAs modulate hematopoietic lineage differentiation
-
Chen CZ, Li L, Lodish HF, Bartel DP. MicroRNAs modulate hematopoietic lineage differentiation. Science 2004; 303: 83-86.
-
(2004)
Science
, vol.303
, pp. 83-86
-
-
Chen, C.Z.1
Li, L.2
Lodish, H.F.3
Bartel, D.P.4
-
24
-
-
33644866985
-
Hematopoietic-specific microRNA expression in human cells
-
Ramkissoon SH, Mainwaring LA, Ogasawara Y, Keyvanfar K, McCoy Jr JP, Sloand EM et al. Hematopoietic-specific microRNA expression in human cells. Leuk Res 2006; 30: 643-647.
-
(2006)
Leuk Res
, vol.30
, pp. 643-647
-
-
Ramkissoon, S.H.1
Mainwaring, L.A.2
Ogasawara, Y.3
Keyvanfar, K.4
McCoy Jr., J.P.5
Sloand, E.M.6
-
25
-
-
79951510234
-
MicroRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors
-
Geisler A, Jungmann A, Kurreck J, Poller W, Katus HA, Vetter R et al. microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors. Gene Therapy 2011; 18: 199-209.
-
(2011)
Gene Therapy
, vol.18
, pp. 199-209
-
-
Geisler, A.1
Jungmann, A.2
Kurreck, J.3
Poller, W.4
Katus, H.A.5
Vetter, R.6
-
26
-
-
67349217385
-
Induction of immune tolerance to FIX following muscular AAV gene transfer is AAV-dose/FIX-level dependent
-
Kelly ME, Zhuo J, Bharadwaj AS, Chao H. Induction of immune tolerance to FIX following muscular AAV gene transfer is AAV-dose/FIX-level dependent. Mol Ther 2009; 17: 857-863.
-
(2009)
Mol Ther
, vol.17
, pp. 857-863
-
-
Kelly, M.E.1
Zhuo, J.2
Bharadwaj, A.S.3
Chao, H.4
-
27
-
-
79953803239
-
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
-
Qiao C, Yuan Z, Li J, He B, Zheng H, Mayer C et al. Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver. Gene Therapy 2011; 18: 403-410.
-
(2011)
Gene Therapy
, vol.18
, pp. 403-410
-
-
Qiao, C.1
Yuan, Z.2
Li, J.3
He, B.4
Zheng, H.5
Mayer, C.6
-
28
-
-
33947721625
-
MiR-181a is an intrinsic modulator of T cell sensitivity and selection
-
Li QJ, Chau J, Ebert PJ, Sylvester G, Min H, Liu G et al. miR-181a is an intrinsic modulator of T cell sensitivity and selection. Cell 2007; 129: 147-161.
-
(2007)
Cell
, vol.129
, pp. 147-161
-
-
Li, Q.J.1
Chau, J.2
Ebert, P.J.3
Sylvester, G.4
Min, H.5
Liu, G.6
-
29
-
-
61749100637
-
Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras
-
Papapetrou EP, Kovalovsky D, Beloeil L, Sant'angelo D, Sadelain M. Harnessing endogenous miR-181a to segregate transgenic antigen receptor expression in developing versus post-thymic T cells in murine hematopoietic chimeras. J Clin Invest 2009; 119: 157-168.
-
(2009)
J Clin Invest
, vol.119
, pp. 157-168
-
-
Papapetrou, E.P.1
Kovalovsky, D.2
Beloeil, L.3
Sant'Angelo, D.4
Sadelain, M.5
-
30
-
-
34249851499
-
MiR-150, a microRNA expressed in mature B and T cells, blocks early B cell development when expressed prematurely
-
Zhou B, Wang S, Mayr C, Bartel DP, Lodish HF. miR-150, a microRNA expressed in mature B and T cells, blocks early B cell development when expressed prematurely. Proc Natl Acad Sci USA 2007; 104: 7080-7085.
-
(2007)
Proc Natl Acad Sci USA
, vol.104
, pp. 7080-7085
-
-
Zhou, B.1
Wang, S.2
Mayr, C.3
Bartel, D.P.4
Lodish, H.F.5
-
31
-
-
85027922313
-
The role of microRNA-150 as a tumor suppressor in malignant lymphoma
-
Watanabe A, Tagawa H, Yamashita J, Teshima K, Nara M, Iwamoto K et al. The role of microRNA-150 as a tumor suppressor in malignant lymphoma. Leukemia 2011; 25: 1324-1334.
-
(2011)
Leukemia
, vol.25
, pp. 1324-1334
-
-
Watanabe, A.1
Tagawa, H.2
Yamashita, J.3
Teshima, K.4
Nara, M.5
Iwamoto, K.6
-
32
-
-
44449108570
-
MicroRNA-mediated control of cell fate in megakaryocyte-erythrocyte progenitors
-
Lu J, Guo S, Ebert BL, Zhang H, Peng X, Bosco J et al. MicroRNA-mediated control of cell fate in megakaryocyte-erythrocyte progenitors. Dev Cell 2008; 14: 843-853.
-
(2008)
Dev Cell
, vol.14
, pp. 843-853
-
-
Lu, J.1
Guo, S.2
Ebert, B.L.3
Zhang, H.4
Peng, X.5
Bosco, J.6
-
33
-
-
79955948510
-
Platelet microRNA-mRNA coexpression profiles correlate with platelet reactivity
-
Nagalla S, Shaw C, Kong X, Kondkar AA, Edelstein LC, Ma L et al. Platelet microRNA-mRNA coexpression profiles correlate with platelet reactivity. Blood 2011; 117: 5189-5197.
-
(2011)
Blood
, vol.117
, pp. 5189-5197
-
-
Nagalla, S.1
Shaw, C.2
Kong, X.3
Kondkar, A.A.4
Edelstein, L.C.5
Ma, L.6
-
34
-
-
70449127230
-
Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors
-
Modlich U, Navarro S, Zychlinski D, Maetzig T, Knoess S, Brugman MH et al. Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol Ther 2009; 17: 1919-1928.
-
(2009)
Mol Ther
, vol.17
, pp. 1919-1928
-
-
Modlich, U.1
Navarro, S.2
Zychlinski, D.3
Maetzig, T.4
Knoess, S.5
Brugman, M.H.6
-
35
-
-
77949424881
-
Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors
-
Maetzig T, Galla M, Brugman MH, Loew R, Baum C, Schambach A. Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors. Gene Therapy 2010; 17: 400-411.
-
(2010)
Gene Therapy
, vol.17
, pp. 400-411
-
-
Maetzig, T.1
Galla, M.2
Brugman, M.H.3
Loew, R.4
Baum, C.5
Schambach, A.6
-
36
-
-
0027228422
-
Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: Concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells
-
Burns JC, Friedmann T, Driever W, Burrascano M, Yee JK. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc Natl Acad Sci USA 1993; 90: 8033-8037.
-
(1993)
Proc Natl Acad Sci USA
, vol.90
, pp. 8033-8037
-
-
Burns, J.C.1
Friedmann, T.2
Driever, W.3
Burrascano, M.4
Yee, J.K.5
-
37
-
-
0034046944
-
Plat-E: An efficient and stable system for transient packaging of retroviruses
-
Morita S, Kojima T, Kitamura T. Plat-E: an efficient and stable system for transient packaging of retroviruses. Gene Therapy 2000; 7: 1063-1066.
-
(2000)
Gene Therapy
, vol.7
, pp. 1063-1066
-
-
Morita, S.1
Kojima, T.2
Kitamura, T.3
-
38
-
-
33646479480
-
Lentiviral vectors pseudotyped with murine ecotropic envelope: Increased biosafety and convenience in preclinical research
-
Schambach A, Galla M, Modlich U, Will E, Chandra S, Reeves L et al. Lentiviral vectors pseudotyped with murine ecotropic envelope: increased biosafety and convenience in preclinical research. Exp Hematol 2006; 34: 588-592.
-
(2006)
Exp Hematol
, vol.34
, pp. 588-592
-
-
Schambach, A.1
Galla, M.2
Modlich, U.3
Will, E.4
Chandra, S.5
Reeves, L.6
-
39
-
-
19944420154
-
A virus-encoded inhibitor that blocks RNA interference in mammalian cells
-
Sullivan CS, Ganem D. A virus-encoded inhibitor that blocks RNA interference in mammalian cells. J Virol 2005; 79: 7371-7379.
-
(2005)
J Virol
, vol.79
, pp. 7371-7379
-
-
Sullivan, C.S.1
Ganem, D.2
-
40
-
-
19444386119
-
Murine hematopoietic stem cells change their surface phenotype during ex vivo expansion
-
Zhang CC, Lodish HF. Murine hematopoietic stem cells change their surface phenotype during ex vivo expansion. Blood 2005; 105: 4314-4320.
-
(2005)
Blood
, vol.105
, pp. 4314-4320
-
-
Zhang, C.C.1
Lodish, H.F.2
|