-
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., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., Marinello, E., Cattaneo, F., Vai, S., Servida, P., Miniero, R., Roncarolo, M.G., and Bordignon, C. (2002). Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 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
Morecki, S.7
Andolfi, G.8
Tabucchi, A.9
Carlucci, F.10
Marinello, E.11
Cattaneo, F.12
Vai, S.13
Servida, P.14
Miniero, R.15
Roncarolo, M.G.16
Bordignon, C.17
-
2
-
-
0035088020
-
Lentivirus vector-mediated hematopoietic stem cell gene transfer of common g-chain cytokine receptor in rhesus macaques
-
An, D.S., Kung, S.K., Bonifacino, A., Wersto, R.P., Metzger, M.E., Agricola, B.A., Mao, S.H., Chen, I.S., and Donahue, R.E. (2001). Lentivirus vector-mediated hematopoietic stem cell gene transfer of common g-chain cytokine receptor in rhesus macaques. J. Virol. 75, 3547-3555.
-
(2001)
J. Virol.
, vol.75
, pp. 3547-3555
-
-
An, D.S.1
Kung, S.K.2
Bonifacino, A.3
Wersto, R.P.4
Metzger, M.E.5
Agricola, B.A.6
Mao, S.H.7
Chen, I.S.8
Donahue, R.E.9
-
3
-
-
10744233606
-
Chance or necessity? Insertional mutagenesis in gene therapy and its consequences [review]
-
Baum, C., Von Kalle, C., Staal, F.J., Li, Z., Fehse, B., Schmidt, M., Weerkamp, F., Karlsson, S., Wagemaker, G., and Williams, D.A. (2004). Chance or necessity? Insertional mutagenesis in gene therapy and its consequences [review]. Mol. Ther. 9, 5-13.
-
(2004)
Mol. Ther.
, vol.9
, pp. 5-13
-
-
Baum, C.1
Von Kalle, C.2
Staal, F.J.3
Li, Z.4
Fehse, B.5
Schmidt, M.6
Weerkamp, F.7
Karlsson, S.8
Wagemaker, G.9
Williams, D.A.10
-
4
-
-
20844445621
-
Cyclosporine inhibits the development of green fluorescent protein (GFP)-specific immune responses after transplantation of GFP-expressing hematopoietic repopulating cells in dogs
-
Beagles, K.E., Peterson, L., Zhang, X., Morris, J., and Kiem, H.-P. (2005). Cyclosporine inhibits the development of green fluorescent protein (GFP)-specific immune responses after transplantation of GFP-expressing hematopoietic repopulating cells in dogs. Hum. Gene Ther. 16, 725-733.
-
(2005)
Hum. Gene Ther.
, vol.16
, pp. 725-733
-
-
Beagles, K.E.1
Peterson, L.2
Zhang, X.3
Morris, J.4
Kiem, H.-P.5
-
5
-
-
34250851635
-
Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells
-
Beard, B.C., Dickerson, D., Beebe, K., Gooch, C., Fletcher, J., Okbinoglu, T., Miller, D.G., Jacobs, M.A., Kaul, R., Kiem, H.-P., and Trobridge, G.D. (2007a). Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells. Mol. Ther. 15, 1356-1365.
-
(2007)
Mol. Ther.
, vol.15
, pp. 1356-1365
-
-
Beard, B.C.1
Dickerson, D.2
Beebe, K.3
Gooch, C.4
Fletcher, J.5
Okbinoglu, T.6
Miller, D.G.7
Jacobs, M.A.8
Kaul, R.9
Kiem, H.-P.10
Trobridge, G.D.11
-
6
-
-
34249804819
-
Unique integration profiles in a canine model of long-term re-populating cells transduced with gammaretrovirus, lentivirus, and foamy virus
-
Beard, B.C., Keyser, K.A., Trobridge, G.D., Peterson, L.J., Miller, D.G., Jacobs, M., Kaul, R., and Kiem, H.-P. (2007b). Unique integration profiles in a canine model of long-term re-populating cells transduced with gammaretrovirus, lentivirus, and foamy virus. Hum. Gene Ther. 18, 423-434.
-
(2007)
Hum. Gene Ther.
, vol.18
, pp. 423-434
-
-
Beard, B.C.1
Keyser, K.A.2
Trobridge, G.D.3
Peterson, L.J.4
Miller, D.G.5
Jacobs, M.6
Kaul, R.7
Kiem, H.-P.8
-
7
-
-
36249015420
-
Gene delivery by lentivirus vectors [review]
-
Cockrell, A.S., and Kafri, T. (2007). Gene delivery by lentivirus vectors [review]. Mol. Biotechnol. 36, 184-204.
-
(2007)
Mol. Biotechnol.
, vol.36
, pp. 184-204
-
-
Cockrell, A.S.1
Kafri, T.2
-
8
-
-
35148817461
-
Gene therapy clinical trials worldwide to 2007: An update [review]
-
Edelstein, M.L., Abedi, M.R., and Wixon, J. (2007). Gene therapy clinical trials worldwide to 2007: An update [review]. J. Gene Med. 9, 833-842.
-
(2007)
J. Gene Med.
, vol.9
, pp. 833-842
-
-
Edelstein, M.L.1
Abedi, M.R.2
Wixon, J.3
-
9
-
-
0037129435
-
Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
-
DOI 10.1056/NEJMoa012616
-
Hacein-Bey-Abina, S., Le Deist, F., Carlier, F., Bouneaud, C., Hue, C., De Villartay, J.P., Thrasher, A.J., Wulffraat, N., Sor-ensen, R., Dupuis-Girod, S., Fischer, A., Davies, E.G., Kuis, W., Leiva, L., and Cavazzana-Calvo, M. (2002). Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N. Engl. J. Med. 346, 1185-1193. (Pubitemid 34984579)
-
(2002)
New England Journal of Medicine
, vol.346
, Issue.16
, pp. 1185-1193
-
-
Hacein-Bey-Abina, S.1
Le Deist, F.2
Carlier, F.3
Bouneaud, C.4
Hue, C.5
De Villartay, J.-P.6
Thrasher, A.J.7
Wulffraat, N.8
Sorensen, R.9
Dupuis-Girod, S.10
Fischer, A.11
Graham Davies, E.12
Kuis, W.13
Leiva, L.14
Cavazzana-Calvo, M.15
-
10
-
-
0142084745
-
LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 [erratum appears in Science 2003;302:568]
-
Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M.P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C.S., Pawliuk, R., Morillon, E., Sorensen, R., Forster, A., Fraser, P., Cohen, J.I., De Saint Basile, G., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Ma-cintyre, E., Sigaux, F., Soulier, J., Leiva, L.E., Wissler, M., Prinz, C., Rabbitts, T.H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M. (2003a). LMO2-associated clonal T cell proliferation
-
(2003)
Science
, vol.302
, pp. 415-419
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
McCormack, M.P.4
Wulffraat, N.5
Leboulch, P.6
Lim, A.7
Osborne, C.S.8
Pawliuk, R.9
Morillon, E.10
Sorensen, R.11
Forster, A.12
Fraser, P.13
Cohen, J.I.14
De Saint Basile, G.15
Alexander, I.16
Wintergerst, U.17
Frebourg, T.18
Aurias, A.19
Stoppa-Lyonnet, D.20
Romana, S.21
Radford-Weiss, I.22
Gross, F.23
Valensi, F.24
Delabesse, E.25
Ma-Cintyre, E.26
Sigaux, F.27
Soulier, J.28
Leiva, L.E.29
Wissler, M.30
Prinz, C.31
Rabbitts, T.H.32
Le Deist, F.33
Fischer, A.34
Cavazzana-Calvo, M.35
more..
-
11
-
-
0037448352
-
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
-
Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., Le Deist, F., Wulffraat, N., McIntyre, E., Radford, I., Villeval, J.L., Fraser, C.C., Cavazzana-Calvo, M., and Fischer, A. (2003b). A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348, 255-256.
-
(2003)
N. Engl. J. Med.
, vol.348
, pp. 255-256
-
-
Hacein-Bey-Abina, S.1
Von Kalle, C.2
Schmidt, M.3
Le Deist, F.4
Wulffraat, N.5
McIntyre, E.6
Radford, I.7
Villeval, J.L.8
Fraser, C.C.9
Cavazzana-Calvo, M.10
Fischer, A.11
-
12
-
-
51349090473
-
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
-
Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., Soulier, J., Lim, A., Morillon, E., Clappier, E., Caccavelli, L., Delabesse, E., Beldjord, K., Asnafi, V., Macintyre, E., Dal Cortivo, L., Rad-ford, I., Brousse, N., Sigaux, F., Moshous, D., Hauer, J., Bork-hardt, A., Belohradsky, B.H., Wintergerst, U., Velez, M.C., Leiva, L., Sorensen, R., Wulffraat, N., Blanche, S., Bushman, F.D., Fischer, A., and Cavazzana-Calvo, M. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132-3142.
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 3132-3142
-
-
Hacein-Bey-Abina, S.1
Garrigue, A.2
Wang, G.P.3
Soulier, J.4
Lim, A.5
Morillon, E.6
Clappier, E.7
Caccavelli, L.8
Delabesse, E.9
Beldjord, K.10
Asnafi, V.11
MacIntyre, E.12
Dal Cortivo, L.13
Rad-Ford, I.14
Brousse, N.15
Sigaux, F.16
Moshous, D.17
Hauer, J.18
Bork-Hardt, A.19
Belohradsky, B.H.20
Wintergerst, U.21
Velez, M.C.22
Leiva, L.23
Sorensen, R.24
Wulffraat, N.25
Blanche, S.26
Bushman, F.D.27
Fischer, A.28
Cavazzana-Calvo, M.29
more..
-
13
-
-
0036846846
-
Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons
-
Horn, P.A., Morris, J.C., Bukovsky, A.A., Andrews, R.G., Nal-dini, L., Kurre, P., and Kiem, H.-P. (2002). Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons. Gene Ther. 9, 1464-1471.
-
(2002)
Gene Ther.
, vol.9
, pp. 1464-1471
-
-
Horn, P.A.1
Morris, J.C.2
Bukovsky, A.A.3
Andrews, R.G.4
Nal-Dini, L.5
Kurre, P.6
Kiem, H.-P.7
-
14
-
-
2342478423
-
Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol
-
Horn, P.A., Keyser, K.A., Peterson, L.J., Neff, T., Thomasson, B.M., Thompson, J., and Kiem, H.-P. (2004). Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood 103, 3710-3716.
-
(2004)
Blood
, vol.103
, pp. 3710-3716
-
-
Horn, P.A.1
Keyser, K.A.2
Peterson, L.J.3
Neff, T.4
Thomasson, B.M.5
Thompson, J.6
Kiem, H.-P.7
-
15
-
-
51349158298
-
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
-
Howe, S.J., Mansour, M.R., Schwarzwaelder, K., Bartholomae, C., Hubank, M., Kempski, H., Brugman, M.H., Pike-Overzet, K., Chatters, S.J., De Ridder, D., Gilmour, K.C., Adams, S., Thornhill, S.I., Parsley, K.L., Staal, F.J., Gale, R.E., Linch, D.C., Bayford, J., Brown, L., Quaye, M., Kinnon, C., Ancliff, P., Webb, D.K., Schmidt, M., Von Kalle, C., Gaspar, H.B., and Thrasher, A.J. (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 118, 3143-3150.
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 3143-3150
-
-
Howe, S.J.1
Mansour, M.R.2
Schwarzwaelder, K.3
Bartholomae, C.4
Hubank, M.5
Kempski, H.6
Brugman, M.H.7
Pike-Overzet, K.8
Chatters, S.J.9
De Ridder, D.10
Gilmour, K.C.11
Adams, S.12
Thornhill, S.I.13
Parsley, K.L.14
Staal, F.J.15
Gale, R.E.16
Linch, D.C.17
Bayford, J.18
Brown, L.19
Quaye, M.20
Kinnon, C.21
Ancliff, P.22
Webb, D.K.23
Schmidt, M.24
Von Kalle, C.25
Gaspar, H.B.26
Thrasher, A.J.27
more..
-
16
-
-
69949164575
-
Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis
-
Kustikova, O.S., Schiedlmeier, B., Brugman, M.H., Stahlhut, M., Bartels, S., Li, Z., and Baum, C. (2009). Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis. Mol. Ther. 17, 1537-1547.
-
(2009)
Mol. Ther.
, vol.17
, pp. 1537-1547
-
-
Kustikova, O.S.1
Schiedlmeier, B.2
Brugman, M.H.3
Stahlhut, M.4
Bartels, S.5
Li, Z.6
Baum, C.7
-
17
-
-
34548661056
-
Second malignancies after allogeneic hematopoietic cell transplantation [review]
-
Lowe, T., Bhatia, S., and Somlo, G. (2007). Second malignancies after allogeneic hematopoietic cell transplantation [review]. Biol. Blood Marrow Transplant. 13, 1121-1134.
-
(2007)
Biol. Blood Marrow Transplant.
, vol.13
, pp. 1121-1134
-
-
Lowe, T.1
Bhatia, S.2
Somlo, G.3
-
18
-
-
67349120266
-
Transcrip-tional enhancers induce insertional gene deregulation independently from the vector type and design
-
Maruggi, G., Porcellini, S., Facchini, G., Perna, S.K., Cattoglio, C., Sartori, D., Ambrosi, A., Schambach, A., Baum, C., Bonini, C., Bovolenta, C., Mavilio, F., and Recchia, A. (2009). Transcrip-tional enhancers induce insertional gene deregulation independently from the vector type and design. Mol. Ther. 17, 851-856.
-
(2009)
Mol. Ther.
, vol.17
, pp. 851-856
-
-
Maruggi, G.1
Porcellini, S.2
Facchini, G.3
Perna, S.K.4
Cattoglio, C.5
Sartori, D.6
Ambrosi, A.7
Schambach, A.8
Baum, C.9
Bonini, C.10
Bovolenta, C.11
Mavilio, F.12
Recchia, A.13
-
19
-
-
58149469932
-
Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells
-
Moreno-Carranza, B., Gentsch, M., Stein, S., Schambach, A., Santilli, G., Rudolf, E., Ryser, M.F., Haria, S., Thrasher, A.J., Baum, C., Brenner, S., and Grez, M. (2009). Transgene optimization significantly improves SIN vector titers, gp91phox expression and reconstitution of superoxide production in X-CGD cells. Gene Ther. 16, 111-118.
-
(2009)
Gene Ther.
, vol.16
, pp. 111-118
-
-
Moreno-Carranza, B.1
Gentsch, M.2
Stein, S.3
Schambach, A.4
Santilli, G.5
Rudolf, E.6
Ryser, M.F.7
Haria, S.8
Thrasher, A.J.9
Baum, C.10
Brenner, S.11
Grez, M.12
-
20
-
-
33645734405
-
Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
-
Ott, M.G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kuhlcke, K., Schilz, A., Kunkel, H., Naundorf, S., Brinkmann, A., Deichmann, A., Fischer, M., Ball, C., Pilz, I., Dunbar, C., Du, Y., Jenkins, N.A., Copeland, N.G., Luthi, U., Hassan, M., Thrasher, A.J., Hoelzer, D., Von Kalle, C., Seger, R., and Grez, M. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 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
Glimm, H.7
Kuhlcke, K.8
Schilz, A.9
Kunkel, H.10
Naundorf, S.11
Brinkmann, A.12
Deichmann, A.13
Fischer, M.14
Ball, C.15
Pilz, I.16
Dunbar, C.17
Du, Y.18
Jenkins, N.A.19
Copeland, N.G.20
Luthi, U.21
Hassan, M.22
Thrasher, A.J.23
Hoelzer, D.24
Von Kalle, C.25
Seger, R.26
Grez, M.27
more..
-
21
-
-
34547807068
-
Foamy virus vectors: An awaited alternative to gammaretro-and lentiviral vectors [review]
-
Rethwilm, A. (2007). Foamy virus vectors: An awaited alternative to gammaretro-and lentiviral vectors [review]. Curr. Gene Ther. 7, 261-271.
-
(2007)
Curr. Gene Ther.
, vol.7
, pp. 261-271
-
-
Rethwilm, A.1
-
22
-
-
36749052217
-
High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR
-
Schmidt, M., Schwarzwaelder, K., Bartholomae, C., Zaoui, K., Ball, C., Pilz, I., Braun, S., Glimm, H., and Von Kalle, C. (2007). High-resolution insertion-site analysis by linear amplification-mediated PCR (LAM-PCR). Nat. Methods 4, 1051-1057.
-
(2007)
Nat. Methods
, vol.4
, pp. 1051-1057
-
-
Schmidt, M.1
Schwarzwaelder, K.2
Bartholomae, C.3
Zaoui, K.4
Ball, C.5
Pilz, I.6
Braun, S.7
Glimm, H.8
Von Kalle, C.9
-
23
-
-
0030694549
-
Gene transfer into haemo-poietic cells [review]
-
Stocking, C., and Baum, C. (1997). Gene transfer into haemo-poietic cells [review]. Baillieres Clin. Haematol. 10, 445-465.
-
(1997)
Baillieres Clin. Haematol.
, vol.10
, pp. 445-465
-
-
Stocking, C.1
Baum, C.2
-
24
-
-
29244441185
-
Hemato-poietic stem cell transduction and amplification in large animal models
-
Trobridge, G., Beard, B.C., and Kiem, H.-P. (2005). Hemato-poietic stem cell transduction and amplification in large animal models. Hum. Gene Ther. 16, 1355-1366.
-
(2005)
Hum. Gene Ther.
, vol.16
, pp. 1355-1366
-
-
Trobridge, G.1
Beard, B.C.2
Kiem, H.-P.3
-
25
-
-
41849142699
-
High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector
-
Zhang, X.-B., Beard, B.C., Trobridge, G.D., Wood, B.L., Sale, G.E., Sud, R., Humphries, R.K., and Kiem, H.-P. (2008). High incidence of leukemia in large animals after stem cell gene therapy with a HOXB4-expressing retroviral vector. J. Clin. Invest. 118, 1502-1510.
-
(2008)
J. Clin. Invest.
, vol.118
, pp. 1502-1510
-
-
Zhang, X.-B.1
Beard, B.C.2
Trobridge, G.D.3
Wood, B.L.4
Sale, G.E.5
Sud, R.6
Humphries, R.K.7
Kiem, H.-P.8
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