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3. LMO2-Associated Clonal T Cell Proliferation in Two Patients after Gene Therapy for SCID-X1

Author

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., Alexander, I., Wintergerst, U., Frebourg, T., Aurias, A., Stoppa-Lyonnet, D., Romana, S., Radford-Weiss, I., Gross, F., Valensi, F., Delabesse, E., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L. E., Wissler, M., Prinz, C., Rabbitts, T. H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M.

Published
2003

4. Autoren

Author

Andrié, R.P., primary, Angenendt, J., additional, Aubin, H., additional, Baldus, S., additional, Balzer, J., additional, Baltzer, J., additional, Behr, J., additional, Benz, Marcus, additional, Berger, M., additional, Blank, N., additional, Blind, E., additional, Buchfelder, M., additional, Burk, A., additional, Detter, C., additional, Diel, R., additional, Diener, H.-C., additional, Domschke, W., additional, Doppler, K., additional, Ellinger, J., additional, Epple, H.-J., additional, Erbel, R., additional, Fichtner-Feigl, S., additional, Fischereder, M., additional, Forsting, M., additional, Friedrich, M., additional, Frommberger, U., additional, Frye, B.C., additional, Goebel, F.-D., additional, Grabitz, K., additional, Groeneveld, A., additional, Gundling, F., additional, Gutt, C.N., additional, Haidl, G., additional, Hartmann, U., additional, Hausteiner-Wiehle, C., additional, Herpertz-Dahlmann, B., additional, Heuß, D., additional, Heußner, P., additional, Hiller, E., additional, Hohenfellner, U., additional, Honegger, J., additional, Hornung, T., additional, Jansen, F., additional, Jelinek, T., additional, Jordan, B., additional, Keck, C., additional, Kelm, M., additional, Kilian, A., additional, Kleindienst, A., additional, Kölbl, H., additional, Kreis, M.E., additional, Kroener-Herwig, B., additional, Külz, A.K., additional, Lammert, F., additional, Lichtenberg, A., additional, Limmroth, V., additional, Lindner, K., additional, Loddenkemper, R., additional, Lorenz, H.-M., additional, Lügering, N., additional, Mallmann, P., additional, Max, R., additional, Merle, U., additional, Mellert, F., additional, Michels, G., additional, Müller, R.-U., additional, Müller, S.C., additional, Müller-Lissner, S., additional, Müller-Quernheim, J., additional, Neuhof, C., additional, Parhofer, K.G., additional, Petri, E., additional, Pfeiffer, T., additional, Pilatz, Adrian, additional, Pizarro, C., additional, Probst, C., additional, Rascher, W., additional, Raue, F., additional, Reindl, C., additional, Riedner, C., additional, Riemann, D., additional, Röckelein, V., additional, Sandmann, W., additional, Sauerbruch, T., additional, Schahab, N., additional, Schaper, A., additional, Schepp, W., additional, Schliep, S., additional, Schnürch, H.-G., additional, Schölmerich, J., additional, Schönermarck, Ulf, additional, Schopohl, J., additional, Schuchert, A., additional, Schulze-Koops, H., additional, Schupp, J., additional, Segerer, S.E., additional, Senninger, N., additional, Seybold, U., additional, Siegmund, B., additional, Skowasch, D., additional, Sommer, C., additional, Stahl, M., additional, Stallmach, A., additional, Sticherling, M., additional, Stremmel, W., additional, Sure, U., additional, Tacke, F., additional, Vloet, T., additional, Voderholzer, U., additional, von Kodolitsch, Y., additional, Wagenlehner, F., additional, Wahlers, T., additional, Weber, L.T., additional, Weidner, W., additional, Weis-Müller, B.T., additional, Werner, N., additional, Wintergerst, U., additional, Wolf, Gunter, additional, Ziegler, D., additional, Zierz, S., additional, and Zimmermann, Th., additional

Published
2019
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6. Pneumonien

Author

Unnewehr, M., primary, Schaaf, B., additional, and Wintergerst, U., additional

Published
2018
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7. Autoren

Author

Albers, P., primary, Andrié, R.P., additional, Angenendt, J., additional, Appenrodt, B., additional, Arkudas, A., additional, Aubin, H., additional, Autschbach, R., additional, Baldus, S., additional, Balzer, J., additional, Baltzer, J., additional, Behr, J., additional, Behrens, A., additional, Benz, Marcus, additional, Berger, M., additional, Berr, F., additional, Bischoff, S.C., additional, Böhm, A., additional, Böhm, M., additional, Bokemeyer, D., additional, Borowitzka, F., additional, Brüwer, M., additional, Buchfelder, M., additional, Burk, A., additional, Caca, K., additional, Daum, S., additional, Detter, C., additional, Diener, H.-C., additional, Domschke, W., additional, Doppler, K., additional, Elger, C.E., additional, Ell, C., additional, Ellinger, J., additional, Epple, H.-J., additional, Esser, B., additional, Ewen, Sebastian, additional, Fibbe, C., additional, Fichtner-Feigl, S., additional, Fiegel, P., additional, Fischbach, W., additional, Fischereder, M., additional, Fölsch, U.R., additional, Forsting, M., additional, Frey, N., additional, Freys, S.M., additional, Friedrich, M., additional, Frieling, T., additional, Frommberger, U., additional, Frühmorgen, P., additional, Geidel, S., additional, Germer, C.-T., additional, Goebel, F.-D., additional, Goetzenich, A., additional, Grabitz, K., additional, Greetfeld, M., additional, Groeneveld, A., additional, Groha, P.K., additional, Gundling, F., additional, Gutt, C.N., additional, Haak, T., additional, Hartmann, U., additional, Hauner, H., additional, Hausteiner-Wiehle, C., additional, Henningsen, P., additional, Herpertz-Dahlmann, B., additional, Heuß, D., additional, Heußner, P., additional, Hoffmann, J., additional, Hohenfellner, U., additional, Horch, R.E., additional, Hüll, M., additional, John, S., additional, Jordan, B., additional, Jurowich, C., additional, Käberich, A., additional, Katus, H., additional, Keck, C., additional, Kelm, M., additional, Kiehne, K., additional, Kilian, Adrienne, additional, Kindermann, I., additional, Kleindienst, A., additional, Klingel, K., additional, von Kodolitsch, Y., additional, Kölbl, H., additional, Koletzko, S., additional, Kollmar, O., additional, Konstantinidis, S., additional, Kramer, H.J., additional, Kreis, M.E., additional, Kroener-Herwig, B., additional, Kurschat, C., additional, Lammert, F., additional, Lamprecht, G., additional, Langosch, J.M., additional, Layer, P., additional, Leifeld, L., additional, Lichtenberg, A., additional, Lindner, K., additional, Link, A., additional, Lügering, N., additional, Lusch, Achim, additional, Marth, T., additional, Merle, U., additional, Mellert, F., additional, Meyer-Marcotty, M., additional, Michels, G., additional, Middeke, J.M., additional, Möddel, G., additional, Mössner, J., additional, Müller, A., additional, Müller, J.L., additional, Müller, R.-U., additional, Müller, S.C., additional, Müller-Lissner, S., additional, Müller-Werdan, U., additional, Neubrand, M., additional, Neuhof, C., additional, Parhofer, K.G., additional, Petri, E., additional, Pfeiffer, T., additional, Pilatz, Adrian, additional, Pizarro, C., additional, Pohle, T., additional, Polykandriotis, E., additional, Probst, C., additional, Rascher, W., additional, Rauch, B., additional, Reuss-Borst, M., additional, Riedner, C., additional, Rietschel, E., additional, Röckelein, V., additional, Rüster, Ch., additional, Ruß, M., additional, Sailer, M., additional, Sandmann, W., additional, Sauerbruch, T., additional, Schaaf, B., additional, Schaefer, C.A., additional, Schahab, N., additional, Schaper, A., additional, Schetelig, J., additional, Schmitz, M., additional, Schnürch, H.-G., additional, Schölmerich, J., additional, Schönermarck, Ulf, additional, Schrickel, J.W., additional, Schuchert, A., additional, Schunkert, H., additional, Segerer, S.E., additional, Senninger, N., additional, Seybold, U., additional, Siegmund, B., additional, Skowasch, D., additional, Sommer, C., additional, Spengler, U., additional, Stahl, M., additional, Stallmach, A., additional, Stolpmann, G., additional, Stremmel, W., additional, Sure, U., additional, Tacke, F., additional, te Wildt, B.T., additional, Trinkler, P., additional, Tröger, H., additional, Unnewehr, M., additional, Vloet, T., additional, Voderholzer, U., additional, Vogt, P.M., additional, von Scheidt, W., additional, Wagenlehner, F., additional, Wahlers, T., additional, Weber, L.T., additional, Wehrmann, T., additional, Weis-Müller, B.T., additional, Werdan, K., additional, Wintergerst, U., additional, Wittwer, T., additional, Wolf, Gunter, additional, Wölfling, K., additional, Wolkersdörfer, G., additional, Ziegler, D., additional, and Zierz, S., additional

Published
2018
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8. Die Untersuchung des Kindes

Author

Rosenecker, J., Priessmann, H., Huppmann, M., Schmidt, H., Arnold, B., Leunig, A., Rudolph, G., Döhlemann, C., Lang, T., Heimkes, B., Enders, A., Giese, R., Bidlingmaier, C., Kurnik, K., Wintergerst, U., Rosenecker, Josef, editor, and Schmidt, Heinrich, editor

Published
2008
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19. Gendiagnostische Forschung an Kindern in Österreich: Spagat zwischen Meilensteinen der Medizin und Mühlsteinen der Bürokratie

Author

Zenz, W., Klobassa, D.S., Sonnleitner, A., Binder, A., Sellner, A., Sperl, M., Wintergerst, U., Huemer, C., Ausserer, B., Stelzl, W., Kaulfersch, W., Grigorow, I., Biebl, A., Wimmer, A., Ortner, D., Emhofer, J., Birnbacher, R., Mostafa, G., Ihm, U., Keck, B., Farr, S., Jaros, Z., Zaunschirm, H.A., Weingarten, C., Glennie, L., van Leeuwen, E., and Levin, M.

Published
2014
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23. Sepsis

Author

Löscher, T., primary, Wintergerst, U., additional, and Nicolai, T., additional

Published
2015
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45. Autorenverzeichnis

Author

Domschke, W., primary, Berger, M., additional, Hohenberger, W., additional, Meinertz, T., additional, Vogelmeier, C., additional, Domschke, W., additional, Sauerbruch, T., additional, Kramer, H.J., additional, Müller, S.C., additional, Serve, H., additional, Weber, M.M., additional, Göke, B., additional, Kalden, J.R., additional, Manger, B., additional, Rascher, W., additional, Appenrodt, B., additional, Atta, J., additional, Auernhammer, C., additional, Autenrieth, I.B., additional, Avenhaus, W., additional, Backmund, M., additional, Bausewein, C., additional, Behr, J., additional, Behrens, A., additional, Berner, R., additional, Berr, F., additional, Blank, N., additional, Blind, E., additional, Bockhorn, M., additional, Bokemeyer, D., additional, Böhm, M., additional, Bönner, G., additional, Borasio, G.D., additional, Bork, K., additional, Braun, J., additional, Bruch, H.-P., additional, Brümmendorf, T.H., additional, Brunnberg, U., additional, Brüwer, M., additional, Büchler, M.W., additional, Detter, C., additional, Diederich, S., additional, Diehm, C., additional, Distler, J., additional, Domagk, D., additional, Dörner, T., additional, Dörr, H.-G., additional, Dralle, H., additional, Dreyling, M., additional, Ehlebracht-König, I., additional, Ell, C., additional, Enzensberger, W., additional, Epple, H.-J., additional, Fassnacht, M., additional, Feußner, H., additional, Fiegel, P., additional, Fisang, C., additional, Filipas, D., additional, Fischbach, W., additional, Flamme, C.H., additional, Floege, J., additional, Fölsch, U.R., additional, Fottner, C., additional, Frank, W., additional, Frey, N., additional, Friese, K., additional, Frilling, A., additional, Fröhner, M., additional, Frühmorgen, P., additional, Galle, P.R., additional, Geidel, S., additional, Genth, E., additional, Gingelmaier, A., additional, Goebel, F.-D., additional, Gökbuget, N., additional, Göke, R., additional, Grabitz, K., additional, Grünke, M., additional, Hahner, S., additional, Handrick, W., additional, Hasslacher, C., additional, Heidbreder, E., additional, Heindel, W., additional, Heinemann, V., additional, Heitmann, J., additional, Heiß, H.W., additional, Hof, H., additional, Hering, L., additional, Hiller, E., additional, Hirner, A., additional, Hofmann, W.-K., additional, Holler, E., additional, Hölscher, A.H., additional, Holtmann, G., additional, Hölzen, J., additional, Honegger, J., additional, Hörle, S., additional, Hörmann, K., additional, Hörmann, R., additional, Hornke, I., additional, Huber, R.M., additional, Hübner, J., additional, Hummel, R., additional, Irmscher, S., additional, Jelinek, T., additional, Jonas, S., additional, Jost, E., additional, Jung, H.H., additional, Kahaly, G.J., additional, Karaus, M., additional, Katsoulis, S., additional, Katus, H., additional, Kessler, H.P., additional, Kiehne, K., additional, Kiess, W., additional, Kindermann, M., additional, von Kodolitsch, Y., additional, Köhler, H., additional, Köhler, L., additional, Köhler, M., additional, Kohne, E., additional, Kolb, H.-J., additional, Köninger, J., additional, Koop, K., additional, Köster, R., additional, Kötter, I., additional, Kramer, H.J.J., additional, Kremer, B., additional, Kroll, P., additional, Kuipers, J.G., additional, Lammert, F., additional, Langer, M., additional, Laukötter, M., additional, Lehnert, H., additional, Lembcke, B., additional, Lerch, M.M., additional, Liebe, S., additional, Lieber, A., additional, Loddenkemper, R., additional, Löhr, M., additional, Lorenz, H.-M., additional, Lorenz, J., additional, Löscher, T., additional, Luster, M., additional, Lux, G., additional, Mann, K., additional, Mayerle, J., additional, Merle, U., additional, Meyer, H.-J., additional, Möbius, C., additional, Moehler, M., additional, Mönnikes, H., additional, Mössner, J., additional, Müller, S.A., additional, Musholt, T.J., additional, Nattermann, J., additional, Neubrand, M., additional, Neuhaus, P., additional, Neundörfer, B., additional, Nicolai, T., additional, Nolde, J., additional, Olschewski, H., additional, Ostermeyer, J., additional, Ott, C., additional, Pahernik, S., additional, Pankratius, U., additional, Parhofer, K.G., additional, Passlick, B., additional, Pech, O., additional, Pfaffenbach, B., additional, Pfeiffer, T., additional, Pilatz, A., additional, Pohle, T., additional, Pohl-Koppe, A., additional, Ponto, Katharina A., additional, Prange, H., additional, Pruß, A., additional, Rädle, J., additional, Rauch, B., additional, Raue, F., additional, Reichel, C., additional, Reindl, C., additional, Reißfelder, C., additional, Rendl, Dipl.-Phys. J., additional, Rietschel, E., additional, Rijcken, E., additional, Roos, R., additional, Rudofsky†, G., additional, Samtleben, W., additional, Sandmann, W., additional, Sauter, G., additional, Schaal, K.P., additional, Schaefer, J.R., additional, Schäfer-Graf, U., additional, Schepp, W., additional, Schlemmer, M., additional, Schliep, S., additional, Schmidt, H., additional, Schmied, B., additional, Schmiegel, W., additional, Schießl, A., additional, Schmid, A., additional, Schneider, A., additional, Schneider, T., additional, Schölmerich, J., additional, Scholz, H., additional, Schönermarck, U., additional, Schopohl, J., additional, Schrezenmeier, H., additional, Schulze-Koops, H., additional, Schuppan, D., additional, Schuster, V., additional, Schüßler, G., additional, Schwandner, O., additional, Schwarz, T.F., additional, Secknus, R., additional, Senninger, N., additional, Sezer, O., additional, Simmen, B.R., additional, Spengler, U., additional, Stabenow-Lohbauer, U., additional, Stebler, R., additional, Steven, D., additional, Sticherling, M., additional, Strauch, U., additional, Stremmel, C., additional, Stremmel, W., additional, Stuck, B.A., additional, Stürz, H., additional, Taube, C., additional, Thulesius, O., additional, Thurau, K., additional, Thüroff, J.W., additional, Tomiak, C., additional, Uhl, W., additional, Vallböhmer, D., additional, Vogel, T., additional, von den Driesch, P., additional, Wagenlehner, F.M.E., additional, Wagner, A., additional, Wagner, U., additional, Weber, K., additional, Weidner, W., additional, Weinke, T., additional, Weis-Müller, B.T., additional, Weiß, M., additional, Willems, S., additional, Wintergerst, U., additional, Wirth, M., additional, Wolkersdörfer, G.W., additional, and Zeitz, M., additional

Published
2011
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46. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

Author

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., Macintyre, E., Sigaux, F., Soulier, J., Leiva, L.E., Wissler, M., Prinz, C., Rabbitts, T.H., Le Deist, F., Fischer, A., and Cavazzana-Calvo, M.

Subjects
Physiological aspects, Complications and side effects, Research, Gene therapy -- Research -- Physiological aspects, Retroviruses -- Research -- Physiological aspects
Abstract

Ex vivo retrovirus-mediated gene transfer into hematopoietic progenitor cells has been shown to be an efficient strategy to correct inherited diseases of the lymphohematopoietic system, provided that a strong selective [...], We have previously shown correction of X-linked severe combined immunodeficiency [SCID-X1, also known as γ chain (γc) deficiency] in 9 out of 10 patients by retrovirus-mediated γc gene transfer into autologous CD34 bone marrow cells. However, almost 3 years after gene therapy, uncontrolled exponential clonal proliferation of mature T cells (with γδ+ or αβ+ T cell receptors) has occurred in the two youngest patients. Both patients' clones showed retrovirus vector integration in proximity to the LMO2 proto-oncogene promoter, leading to aberrant transcription and expression of LMO2. Thus, retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.

Published
2003

47. Empfehlungen zur antiretroviralen Therapie bei HIV-infizierten KindernVollständig überarbeitetes und aktualisiertes Konsensusstatement der Pädiatrischen Arbeitsgemeinschaft Aids (PAAD) und der Deutschen Gesellschaft für Pädiatrische Infektiologie (DGPI): Vollständig überarbeitetes und aktualisiertes Konsensusstatement der Pädiatrischen Arbeitsgemeinschaft Aids (PAAD) und der Deutschen Gesellschaft für Pädiatrische Infektiologie (DGPI)

Author

Niehues, T., Wintergerst, U., Funk, M., Notheis, G., and Für die Konsensusgruppe der Pädiatrischen Arbeitsgemeinschaft Aids (PAAD)

Published
2001
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49. Aktuelle Empfehlungen zur antiretroviralen Therapie bei HIV-infizierten Kindern Konsensus-Statement¶der Deutschen Gesellschaft für Kinderheilkunde und Jugendmedizin,¶der Deutschen Gesellschaft für Pädiatrische Infektiologie und¶der Arbeitsgemeinschaft für Pädiatrische Immunologie: Konsensus-Statement¶der Deutschen Gesellschaft für Kinderheilkunde und Jugendmedizin,¶der Deutschen Gesellschaft für Pädiatrische Infektiologie und¶der Arbeitsgemeinschaft für Pädiatrische Immunologie

Author

Wahn, V., Bialek, R., Böhler, T., Funk, M., Grosch-Wörner, I., Horneff, G., Notheis, G., and Wintergerst, U.

Published
1998
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