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22 results on '"Schilz, Andrea"'

8. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1

Author

Ott, Marion G, Schmidt, Manfred, Schwarzwaelder, Kerstin, Stein, Stefan, Siler, Ulrich, Koehl, Ulrike, Glimm, Hanno, Kuhlcke, Klaus, Schilz, Andrea, Kunkel, Hana, Naundorf, Sonja, Brinkmann, Andrea, Deichmann, Annette, Fischer, Marlene, Ball, Claudia, Pilz, Ingo, Dunbar, Cynthia, Du, Yang, Jenkins, Nancy A, Copeland, Neal G, Luthi, Ursula, Hassan, Moustapha, Thrasher, Adrian J, Hoelzer, Dieter, von Kalle, Christof, Seger, Reinhard, and Grez, Manuel

Abstract

Gene transfer into hematopoietic stem cells has been used successfully for correcting lymphoid but not myeloid immunodeficiencies. Here we report on two adults who received gene therapy after nonmyeloablative bone marrow conditioning for the treatment of X-linked chronic granulomatous disease (X-CGD), a primary immunodeficiency caused by a defect in the oxidative antimicrobial activity of phagocytes resulting from mutations in gp91[sup.phox]. We detected substantial gene transfer in both individuals' neutrophils that lead to a large number of functionally corrected phagocytes and notable clinical improvement. Large-scale retroviral integration site-distribution analysis showed activating insertions in MDS1-EVI1, PRDM16 or SETBP1 that had influenced regulation of long-term hematopoiesis by expanding gene-corrected myelopoiesis three- to four-fold in both individuals. Although insertional influences have probably reinforced the therapeutic efficacy in this trial, our results suggest that gene therapy in combination with bone marrow conditioning can be successfully used to treat inherited diseases affecting the myeloid compartment such as CGD., Author(s): Marion G Ott [1, 16]; Manfred Schmidt [2, 3, 4, 16]; Kerstin Schwarzwaelder [3, 4, 5, 16]; Stefan Stein [6, 16]; Ulrich Siler [7, 16]; Ulrike Koehl [8]; Hanno [...]

Published
2006
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9. Digitale Raumdarstellung

Author

Hoppe, Stephan, Locher, Hubert, Burioni, Matteo, Eikema Hommes, Margriet Van, Pottasch, Carola, Rode, Jan Van, Moor, L. De, Hanschke, Julian, Leipold, Frieder, Lutteroth, Jan-Eric, Grellert, Marc, Drost, Ludger, Grams, Frauke, Hofstetter, Adolf, März, Magdalena, Rehbein, Malte, Sauer, Tomas, Schilz, Andrea, Maxzin, Joerg, Albers, Laura, Ma, Ke, Oberem, Lukas, Scheidt, Thomas, Rahrig, Max, Mayer, Elisabeth, Weil, Lea, Weinand, Kris, Odaker, Thomas, Strackenbrock, Bernhard, and Schwartz, Frithjof

Published
2020
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10. 'Exakt Historisch' im Digitalen? Versuch einer Anleihe

Author

Schilz, Andrea, Vogeler, Georg, and Helling, Patrick

Subjects
Born Digital, DHd2018, Digital Humanities, Quellenkritik, Digitale Quellenkritik, Volkskunde, Vergleichende Kulturwissenschaft, Empirische Kulturwissenschaft, Europäische Ethnologie, Datenkritik, Exakt historische Methode
Abstract

A single abstract from the DHd-2018 Book of Abstracts., Sofern eine editorische Arbeit an dieser Publikation stattgefunden hat, dann bestand diese aus der Eliminierung von Bindestrichen in Überschriften, die aufgrund fehlerhafter Silbentrennung entstanden sind, der Vereinheitlichung von Namen der Autor*innen in das Schema "Nachname, Vorname" und/oder der Trennung von Überschrift und Unterüberschrift durch die Setzung eines Punktes, sofern notwendig., {"references":["https://doi.org/10.5281/zenodo.3684897","https://github.com/DHd-Verband/DHd-Abstracts-2018"]}

Published
2018
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13. Phase I/II Gene Therapy Study for Chronic Granulomatous Disease: Results, Lessons and Perspectives.

Author

Ott, Marion G., primary, Stein, Stefan, additional, Schultze-Strasser, Stephan, additional, Jauch, Anna, additional, Burwinkel, Barbara, additional, Schlegelberger, Brigitte, additional, Goehring, Gudrun, additional, Preiss, Carolin, additional, Kramer, Rolf, additional, Schwarzwaelder, Kerstin, additional, Koehl, Ulrike, additional, Kunkel, Hanna, additional, Kuehlcke, Klaus, additional, Schilz, Andrea, additional, Schmidt, Manfred, additional, von Kalle, Christof, additional, Trasher, Adrian, additional, Hoelzer, Dieter, additional, Seger, Reinhard, additional, and Grez, Manuel, additional

Published
2007
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14. Long-Term Clinical Follow-Up and Safety/Toxicity Analysis in 3 X-CGD Patients Treated by Gene Therapy and Non-Myeloablative Conditioning.

Author

Ott, Marion G., primary, Schmidt, Manfred, additional, Stein, Stefan, additional, Schwarzwaelder, Kerstin, additional, Siler, Ulrich, additional, Koehl, Ulrike, additional, Kuehlcke, Klaus, additional, Schilz, Andrea, additional, Kalle, Christoph v., additional, Hoelzer, Dieter, additional, Seger, Reinhard, additional, and Grez, Manuel, additional

Published
2006
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15. Long-Term Follow-Up of Patients Treated by Gene Therapy for X-Linked Chronic Granulomatous Disease.

Author

Ott, Marion G., primary, Grez, Manuel, additional, Stein, Stefan, additional, Siler, Ulrich, additional, Koehl, Ulrike, additional, Kuehlcke, Klaus, additional, Schmidt, Manfred, additional, Schilz, Andrea, additional, Kunkel, Hana, additional, Naundorf, Sonja, additional, Brinkmann, Andrea, additional, Hassan, Moustapha, additional, von Kalle, Christof, additional, Trasher, Adrian, additional, Seger, Reinhard, additional, and Hoelzer, Dieter, additional

Published
2005
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16. Gene Therapy for X-Linked Chronic Granulomatous Disease.

Author

Ott, Marion G., primary, Stein, Stefan, primary, Koehl, Ulrike, primary, Schilz, Andrea, primary, Kuhlcke, Klaus, primary, Schmidt, Manfred, primary, von Kalle, Christof, primary, Hassan, Moustapha, primary, Siler, Ulrich, primary, Hoelzer, Dieter, primary, Seger, Reinhard, primary, and Grez, Manuel, primary

Published
2004
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21. 336. Correction of Chronic Granulomatous Disease by Gene Therapy

Author

Grez, Manuel, Ott, Marion G., Stein, Stefan, Siler, Ulrich, Koehl, Ulrike, Kunkel, Hana, Schilz, Andrea, Kuehlcke, Klaus, Hoelzer, Dieter, and Seger, Reinhard

Subjects
*GENE therapy, *CHRONIC granulomatous disease
Abstract

An abstract of the article "Correction of Chronic Granulomatous Disease by Gene Therapy," by Manuel Grez, Marion G. Ott, Stefan Stein, Ulrich Siler, Ulrike Koehl, Hana Kunkel, Andrea Schilz, Klaus Kuehlcke, Dieter Hoelzer and Reinhard Seger is presented.

Published
2005
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22. 495. Upscaling of Retroviral Vector Production under GMP Guidelines to Generate High Titer Preparations for Clinical Use

Author

Jung, Sandra, Leyendecker, Peter, Kogelberg, Frank, Wolf, Christian, Schilz, Andrea, Kuehlcke, Klaus, and Naundorf, Sonja

Subjects
*GENE therapy, *CLINICAL trials
Abstract

An abstract of the article "Upscaling of Retroviral Vector Production under GMP Guidelines to Generate High Titer Preparations for Clinical Use," by Sandra Jung, Peter Leyendecker, Frank Kogelberg, Christian Wolf, Andrea Schilz, Klaus Kuehlcke and Sonja Naundorf is presented.

Published
2005
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