C
Cynthia C. Bartholomae
Researcher at German Cancer Research Center
Publications - 47
Citations - 9749
Cynthia C. Bartholomae is an academic researcher from German Cancer Research Center. The author has contributed to research in topics: Genetic enhancement & Viral vector. The author has an hindex of 30, co-authored 47 publications receiving 8835 citations.
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Journal ArticleDOI
Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Nathalie Cartier,Salima Hacein-Bey-Abina,Cynthia C. Bartholomae,Gabor Istvan Veres,Manfred Schmidt,Ina Kutschera,Michel Vidaud,Ulrich Abel,Liliane Dal-Cortivo,Laure Caccavelli,Nizar Mahlaoui,Veronique Kiermer,Denice Mittelstaedt,Céline Bellesme,Najiba Lahlou,François Lefrère,Stéphane Blanche,Muriel Audit,Emmanuel Payen,Philippe Leboulch,Philippe Leboulch,Bruno l’Homme,Pierre Bougnères,Christof von Kalle,Alain Fischer,Marina Cavazzana-Calvo,Patrick Aubourg +26 more
TL;DR: Lentiviral-mediated gene therapy of hematopoietic stem cells can provide clinical benefits in ALD, and progressive cerebral demyelination in the two patients stopped, a clinical outcome comparable to that achieved by allogeneic HCT.
Journal ArticleDOI
Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
Steven J. Howe,Marc R. Mansour,Kerstin Schwarzwaelder,Cynthia C. Bartholomae,Mike Hubank,Helena Kempski,Martijn H. Brugman,Karin Pike-Overzet,S Chatters,Dick de Ridder,Dick de Ridder,Kimberly Gilmour,Stuart Adams,Susannah I. Thornhill,Kathryn L. Parsley,Frank J. T. Staal,Rosemary E. Gale,David C. Linch,Jinhua Bayford,Lucie Brown,Michelle Quaye,Christine Kinnon,Philip Ancliff,David Webb,Manfred Schmidt,Christof von Kalle,H. Bobby Gaspar,Adrian J. Thrasher +27 more
TL;DR: The occurrence of clonal T cell acute lymphoblastic leukemia (T-ALL) promoted by insertional mutagenesis in a completed gene therapy trial of 10 SCID-X1 patients is described and a general toxicity of endogenous gammaretroviral enhancer elements is highlighted.
Journal ArticleDOI
Dissecting the genomic complexity underlying medulloblastoma
David T.W. Jones,Natalie Jäger,Marcel Kool,Thomas Zichner,Barbara Hutter,Marc Sultan,Yoon Jae Cho,Trevor J. Pugh,Volker Hovestadt,Adrian M. Stütz,Tobias Rausch,Hans-Jörg Warnatz,Marina Ryzhova,Sebastian Bender,Dominik Sturm,Sabrina Pleier,Huriye Cin,Elke Pfaff,Laura Sieber,Andrea Wittmann,Marc Remke,Hendrik Witt,Hendrik Witt,Sonja Hutter,Theophilos Tzaridis,Joachim Weischenfeldt,Benjamin Raeder,Meryem Avci,Vyacheslav Amstislavskiy,Marc Zapatka,Ursula D. Weber,Qi Wang,Bärbel Lasitschka,Cynthia C. Bartholomae,Manfred Schmidt,Christof von Kalle,Volker Ast,Chris Lawerenz,Jürgen Eils,Rolf Kabbe,Vladimir Benes,Peter van Sluis,Jan Koster,Richard Volckmann,David Shih,Matthew J. Betts,Robert B. Russell,Simona Coco,Gian Paolo Tonini,Ulrich Schüller,Volkmar Hans,Norbert Graf,Yoo-Jin Kim,Camelia M. Monoranu,Wolfgang Roggendorf,Andreas Unterberg,Christel Herold-Mende,Till Milde,Till Milde,Andreas E. Kulozik,Andreas von Deimling,Andreas von Deimling,Olaf Witt,Olaf Witt,Eberhard Maass,Jochen Rössler,Martin Ebinger,Martin U. Schuhmann,Michael C. Frühwald,Martin Hasselblatt,Nada Jabado,Stefan Rutkowski,André O. von Bueren,Daniel Williamson,Steven C. Clifford,Martin G. McCabe,Martin G. McCabe,V. Peter Collins,Stephan Wolf,Stefan Wiemann,Hans Lehrach,Benedikt Brors,Wolfram Scheurlen,Jörg Felsberg,Guido Reifenberger,Paul A. Northcott,Michael D. Taylor,Matthew Meyerson,Matthew Meyerson,Scott L. Pomeroy,Scott L. Pomeroy,Marie-Laure Yaspo,Jan O. Korbel,Andrey Korshunov,Andrey Korshunov,Roland Eils,Roland Eils,Stefan M. Pfister,Stefan M. Pfister,Peter Lichter +99 more
TL;DR: An integrative deep-sequencing analysis of 125 tumour–normal pairs enhances the understanding of the genomic complexity and heterogeneity underlying medulloblastoma, and provides several potential targets for new therapeutics, especially for Group 3 and 4 patients.
Journal ArticleDOI
Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
Eugenio Montini,Daniela Cesana,Manfred G. Schmidt,Francesca Sanvito,Maurilio Ponzoni,Cynthia C. Bartholomae,Lucia Sergi Sergi,Fabrizio Benedicenti,Alessandro Ambrosi,Clelia Di Serio,Claudio Doglioni,Christof von Kalle,Luigi Naldini +12 more
TL;DR: The results validate a much-needed platform to assess vector safety and provide direct evidence that prototypical lentiviral vectors have low oncogenic potential, highlighting a major rationale for application to gene therapy.
Journal ArticleDOI
Recurrent somatic alterations of FGFR1 and NTRK2 in pilocytic astrocytoma
David T.W. Jones,Barbara Hutter,Natalie Jäger,Andrey Korshunov,Andrey Korshunov,Marcel Kool,Hans-Jörg Warnatz,Thomas Zichner,Sally R. Lambert,Marina Ryzhova,Dong Anh Khuong Quang,Adam M. Fontebasso,Adrian M. Stütz,Sonja Hutter,Marc Zuckermann,Dominik Sturm,Jan Gronych,Bärbel Lasitschka,Sabine Schmidt,Huriye Seker-Cin,Hendrik Witt,Hendrik Witt,Marc Sultan,Meryem Ralser,Paul A. Northcott,Volker Hovestadt,Sebastian Bender,Elke Pfaff,Sebastian Stark,Damien Faury,Jeremy Schwartzentruber,Jacek Majewski,Ursula D. Weber,Marc Zapatka,Benjamin Raeder,Matthias Schlesner,Catherine L. Worth,Cynthia C. Bartholomae,Christof von Kalle,Charles D. Imbusch,S. Radomski,S. Radomski,Chris Lawerenz,Peter van Sluis,Jan Koster,Richard Volckmann,Rogier Versteeg,Hans Lehrach,Camelia M. Monoranu,Beate Winkler,Andreas Unterberg,Christel Herold-Mende,Till Milde,Till Milde,Andreas E. Kulozik,Martin Ebinger,Martin U. Schuhmann,Yoon Jae Cho,Scott L. Pomeroy,Scott L. Pomeroy,Andreas von Deimling,Andreas von Deimling,Olaf Witt,Olaf Witt,Michael D. Taylor,Stephan Wolf,Matthias A. Karajannis,Charles G. Eberhart,Wolfram Scheurlen,Martin Hasselblatt,Keith L. Ligon,Mark W. Kieran,Jan O. Korbel,Marie-Laure Yaspo,Benedikt Brors,Jörg Felsberg,Guido Reifenberger,V. Peter Collins,Nada Jabado,Nada Jabado,Roland Eils,Roland Eils,Peter Lichter +82 more
TL;DR: Recurrent activating mutations in FGFR1 and PTPN11 and new NTRK2 fusion genes in non-cerebellar tumors and new BRAF-activating changes were observed, indicating that pilocytic astrocytoma is predominantly a single-pathway disease.