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adrenoleukodystrophy - therapy (3) 3
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Science, ISSN 0036-8075, 11/2009, Volume 326, Issue 5954, pp. 818 - 823
X-linked adrenoleukodystrophy (ALD) is a severe brain demyelinating disease in boys that is caused by a deficiency in ALD protein, an adenosine... 
Myeloid cells | Genetic vectors | Demyelinating diseases | B lymphocytes | Stem cells | Hematocrit | Cell lines | Gene therapy | Cells | Hematopoietic stem cells | Research Article | HIV VECTORS | SEVERE COMBINED IMMUNODEFICIENCY | INTEGRATION SITE SELECTION | MICROGLIA | BONE-MARROW-TRANSPLANTATION | HUMAN CD34(+) CELLS | MULTIDISCIPLINARY SCIENCES | MOUSE MODEL | NOD/SCID MICE | LONG-TERM ENGRAFTMENT | DIFFERENTIATE | Myeloablative Agonists - therapeutic use | Genetic Therapy | Leukocytes, Mononuclear - metabolism | Microglia - metabolism | Humans | Adrenoleukodystrophy - pathology | Male | Transplantation, Autologous | ATP-Binding Cassette Transporters - genetics | Hematopoiesis | Adrenoleukodystrophy - therapy | Hematopoietic Stem Cells - physiology | Female | Cell Differentiation | Fatty Acids - blood | Hematopoietic Stem Cells - virology | Child | Microglia - cytology | Gene Expression | Transduction, Genetic | Hematopoietic Stem Cell Transplantation | ATP Binding Cassette Transporter, Sub-Family D, Member 1 | HIV-1 - genetics | Virus Integration | Disease Progression | Cell Lineage | Animals | Brain - pathology | Mice | Adrenoleukodystrophy - genetics | Transplantation Conditioning | Genetic Vectors | Usage | Care and treatment | Adrenoleukodystrophy | Patient outcomes | Transplantation | Health aspects | Viruses | Genetic disorders | Neurological disorders | ABCD1 protein | Index Medicus | Life Sciences | Cellular Biology
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 04/2009, Volume 119, Issue 4, pp. 964 - 975
Journal Article
by Kool, Marcel and Jones, David T.W and Jäger, Natalie and Northcott, Paul A and Pugh, Trevor J and Hovestadt, Volker and Piro, Rosario M and Esparza, L. Adriana and Markant, Shirley L and Remke, Marc and Milde, Till and Bourdeaut, Franck and Ryzhova, Marina and Sturm, Dominik and Pfaff, Elke and Stark, Sebastian and Hutter, Sonja and Şeker-Cin, Huriye and Johann, Pascal and Bender, Sebastian and Schmidt, Christin and Schmidt, Sabine and Rausch, Tobias and Shih, David and Reimand, Jüri and Sieber, Laura and Wittmann, Andrea and Linke, Linda and Witt, Olaf and Witt, Hendrik and Weber, Ursula D and Zapatka, Marc and König, Rainer and Beroukhim, Rameen and Bergthold, Guillaume and van Sluis, Peter and Volckmann, Richard and Koster, Jan and Versteeg, Rogier and Wolf, Stephan and Lawerenz, Chris and Bartholomae, Cynthia C and von Kalle, Christof and Unterberg, Andreas and Herold-Mende, Christel and Hofer, Silvia and Kulozik, Andreas E and von Deimling, Andreas and Scheurlen, Wolfram and Felsberg, Jörg and Reifenberger, Guido and Hasselblatt, Martin and Crawford, John R and Grant, Gerald A and Jabado, Nada and Perry, Arie and Cowdrey, Cynthia and Croul, Sydney and Zadeh, Gelareh and Korbel, Jan O and Doz, Francois and Delattre, Olivier and Bader, Gary D and McCabe, Martin G and Collins, V. Peter and Kieran, Mark W and Cho, Yoon-Jae and Pomeroy, Scott L and Brors, Benedikt and Taylor, Michael D and Schüller, Ulrich and Korshunov, Andrey and Eils, Roland and Wechsler-Reya, Robert J and Lichter, Peter and Pfister, Stefan M and ICGC PedBrain Tumor Project
Cancer Cell, ISSN 1535-6108, 03/2014, Volume 25, Issue 3, pp. 393 - 405
Smoothened (SMO) inhibitors recently entered clinical trials for sonic-hedgehog-driven medulloblastoma (SHH-MB). Clinical response is highly variable. To... 
NERVOUS-SYSTEM | TERT PROMOTER MUTATIONS | ONCOLOGY | GROWTH | HEDGEHOG PATHWAY INHIBITOR | ITRACONAZOLE | PEDIATRIC MEDULLOBLASTOMA | CANCER | TUMORS | MOUSE MODELS | ADULT | CELL BIOLOGY | Neoplasm Transplantation | Oncogene Proteins - genetics | Humans | Zinc Finger Protein Gli2 | Child, Preschool | Molecular Sequence Data | Infant | Male | Gene Expression Profiling | Phosphatidylinositol 3-Kinases - metabolism | Biphenyl Compounds - therapeutic use | Promoter Regions, Genetic - genetics | Proto-Oncogene Proteins c-akt - genetics | Tumor Suppressor Protein p53 - genetics | Young Adult | Telomerase - genetics | Hedgehog Proteins - genetics | Base Sequence | Smoothened Receptor | Adult | Female | N-Myc Proto-Oncogene Protein | Patched Receptors | Medulloblastoma - genetics | Nuclear Proteins - genetics | Proto-Oncogene Proteins c-akt - metabolism | Child | Pyridines - therapeutic use | Cerebellar Neoplasms - drug therapy | Repressor Proteins - genetics | DNA Copy Number Variations - genetics | Signal Transduction - genetics | Cerebellar Neoplasms - genetics | Mice, SCID | Phosphatidylinositol 3-Kinases - genetics | DEAD-box RNA Helicases - genetics | Drug Resistance, Neoplasm - genetics | Animals | Adolescent | Receptors, G-Protein-Coupled - antagonists & inhibitors | Mice, Inbred NOD | High-Throughput Nucleotide Sequencing | Mice | Receptors, G-Protein-Coupled - genetics | Medulloblastoma - drug therapy | Kruppel-Like Transcription Factors - genetics | Patched-1 Receptor | Receptors, Cell Surface - genetics | Genetic aspects | Nucleotide sequencing | Analysis | Medulloblastoma | Genomics | DNA sequencing | Index Medicus
Journal Article
by Jones, David T. W and Hutter, Barbara and Jäger, Natalie and Korshunov, Aney and Kool, Marcel and Warnatz, Hans-Jörg and Zichner, Thomas and Lambert, Sally R and Ryzhova, Marina and Quang, Dong Anh Khuong and Fontebasso, Adam M and Stütz, Aian M and Hutter, Sonja and Zuckermann, Marc and Sturm, Dominik and Gronych, Jan and Lasitschka, Bärbel and Schmidt, Sabine and Seker-Cin, Huriye and Witt, Henik and Sultan, Marc and Ralser, Meryem and Northcott, Paul A and Hovestadt, Volker and Bender, Sebastian and Pfaff, Elke and Stark, Sebastian and Faury, Damien and Schwartzentruber, Jeremy and Majewski, Jacek and Weber, Ursula D and Zapatka, Marc and Raeder, Benjamin and Schlesner, Matthias and Worth, Catherine L and Bartholomae, Cynthia C and von Kalle, Christof and Imbusch, Charles D and Radomski, Sylwester and Lawerenz, Chris and van Sluis, Peter and Koster, Jan and Volckmann, Richard and Versteeg, Rogier and Lehrach, Hans and Monoranu, Camelia and Winkler, Beate and Unterberg, Aneas and Herold-Mende, Christel and Milde, Till and Kulozik, Aneas E and Ebinger, Martin and Schuhmann, Martin U and Cho, Yoon-Jae and Pomeroy, Scott L and von Deimling, Aneas and Witt, Olaf and Taylor, Michael D and Wolf, Stephan and Karajannis, Matthias A and Eberhart, Charles G and Scheurlen, Wolfram and Hasselblatt, Martin and Ligon, Keith L and Kieran, Mark W and Korbel, Jan O and Yaspo, Marie-Laure and Brors, Benedikt and Felsberg, Jörg and Reifenberger, Guido and Collins, V. Peter and Jabado, Nada and Eils, Roland and Lichter, Peter and Pfister, Stefan M and Int Canc Genome Consortium and International Cancer Genome Consortium PedBrain Tumor Project and the International Cancer Genome Consortium PedBrain Tumor Project
Nature genetics, ISSN 1061-4036, 2013, Volume 45, Issue 8, pp. 927 - 932
Journal Article
Nature, ISSN 0028-0836, 2014, Volume 510, Issue 7506, pp. 537 - 541
Journal Article
Journal Article
by Jones, David T. W and Jäger, Natalie and Kool, Marcel and Zichner, Thomas and Hutter, Barbara and Sultan, Marc and Cho, Yoon-Jae and Pugh, Trevor J and Hovestadt, Volker and Stütz, Aian M and Rausch, Tobias and Warnatz, Hans-Jörg and Ryzhova, Marina and Bender, Sebastian and Sturm, Dominik and Pleier, Sabrina and Cin, Huriye and Pfaff, Elke and Sieber, Laura and Wittmann, Anea and Remke, Marc and Witt, Henik and Hutter, Sonja and Tzaridis, Theophilos and Weischenfeldt, Joachim and Raeder, Benjamin and Avci, Meryem and Amstislavskiy, Vyacheslav and Zapatka, Marc and Weber, Ursula D and Wang, Qi and Lasitschka, Bärbel and Bartholomae, Cynthia C and Schmidt, Manfred and von Kalle, Christof and Ast, Volker and Lawerenz, Chris and Eils, Jürgen and Kabbe, Rolf and Benes, Vladimir and van Sluis, Peter and Koster, Jan and Volckmann, Richard and Shih, David and Betts, Matthew J and Russell, Robert B and Coco, Simona and Tonini, Gian Paolo and Schüller, Ulrich and Hans, Volkmar and Graf, Norbert and Kim, Yoo-Jin and Monoranu, Camelia and Roggendorf, Wolfgang and Unterberg, Aneas and Herold-Mende, Christel and Milde, Till and Kulozik, Aneas E and von Deimling, Aneas and Witt, Olaf and Maass, Eberhard and Rössler, Jochen and Ebinger, Martin and Schuhmann, Martin U and Frühwald, Michael C and Hasselblatt, Martin and Jabado, Nada and Rutkowski, Stefan and von Bueren, Ané O and Williamson, Dan and Clifford, Steven C and McCabe, Martin G and Collins, V. Peter and Wolf, Stephan and Wiemann, Stefan and Lehrach, Hans and Brors, Benedikt and Scheurlen, Wolfram and Felsberg, Jörg and Reifenberger, Guido and Northcott, Paul A and Taylor, Michael D and Meyerson, Matthew and Pomeroy, Scott L and Yaspo, Marie-Laure and Korbel, Jan O and Korshunov, Aney and Eils, Roland and Pfister, Stefan M and Lichter, Peter
Nature, ISSN 0028-0836, 2012, Volume 488, Issue 7409, pp. 100 - 105
Medulloblastoma is an aggressively growing tumour, arising in the cerebellum or medulla/brain stem. It is the most common malignant brain tumour in children,... 
ACTIVATION | PATHWAY | LANDSCAPE | MULTIDISCIPLINARY SCIENCES | GENES | TETRAPLOID TUMOR-CELLS | TBR1 | MUTATIONS | SUBGROUPS | CANCER | Chromatin - metabolism | Cerebellar Neoplasms - diagnosis | Genomics | Humans | Hedgehog Proteins - metabolism | Wnt Proteins - metabolism | Histone Demethylases - genetics | Aging - genetics | Medulloblastoma - pathology | Cerebellar Neoplasms - pathology | Patched Receptors | Cerebellar Neoplasms - classification | Medulloblastoma - genetics | Neoplasm Proteins - genetics | Nuclear Proteins - genetics | Child | DNA Helicases - genetics | Amino Acid Sequence | Polyploidy | Signal Transduction | Chromosomes, Human - genetics | Medulloblastoma - diagnosis | Mutation Rate | Cerebellar Neoplasms - genetics | Transcription Factors - genetics | DNA-Binding Proteins - genetics | Mutation - genetics | T-Box Domain Proteins - genetics | Genome, Human - genetics | beta Catenin - genetics | DEAD-box RNA Helicases - genetics | Phosphoprotein Phosphatases - genetics | Sequence Analysis, RNA | Cell Transformation, Neoplastic | Oncogene Proteins, Fusion - genetics | High-Throughput Nucleotide Sequencing | Methylation | Medulloblastoma - classification | Patched-1 Receptor | Receptors, Cell Surface - genetics | Medulloblastoma | Physiological aspects | Development and progression | Genetic aspects | Research | Metastasis | Cancer | Index Medicus
Journal Article
by Northcott, Paul A and Lee, Catherine and Zichner, Thomas and Stütz, Aian M and Erkek, Serap and Kawauchi, Daisuke and Shih, David J. H and Hovestadt, Volker and Zapatka, Marc and Sturm, Dominik and Jones, David T. W and Kool, Marcel and Remke, Marc and Cavalli, Florence M. G and Zuyderduyn, Scott and Bader, Gary D and VandenBerg, Scott and Esparza, Lourdes Aiana and Ryzhova, Marina and Wang, Wei and Wittmann, Anea and Stark, Sebastian and Sieber, Laura and Seker-Cin, Huriye and Linke, Linda and Kratochwil, Fabian and Jäger, Natalie and Buchhalter, Ivo and Imbusch, Charles D and Zipprich, Gideon and Raeder, Benjamin and Schmidt, Sabine and Diessl, Nicolle and Wolf, Stephan and Wiemann, Stefan and Brors, Benedikt and Lawerenz, Chris and Eils, Jürgen and Warnatz, Hans-Jörg and Risch, Thomas and Yaspo, Marie-Laure and Weber, Ursula D and Bartholomae, Cynthia C and von Kalle, Christof and Turányi, Eszter and Hauser, Peter and Sanden, Emma and Darabi, Anna and Siesjö, Peter and Sterba, Jaroslav and Zitterbart, Karel and Sumerauer, David and van Sluis, Peter and Versteeg, Rogier and Volckmann, Richard and Koster, Jan and Schuhmann, Martin U and Ebinger, Martin and Grimes, H. Leighton and Robinson, Giles W and Gajjar, Amar and Mynarek, Martin and von Hoff, Katja and Rutkowski, Stefan and Pietsch, Torsten and Scheurlen, Wolfram and Felsberg, Jörg and Reifenberger, Guido and Kulozik, Aneas E and von Deimling, Aneas and Witt, Olaf and Eils, Roland and Gilbertson, Richard J and Korshunov, Aney and Taylor, Michael D and Lichter, Peter and Korbel, Jan O and Wechsler-Reya, Robert J and Pfister, Stefan M and Neurosurgery and Neurokirurgi and Lund University and BioCARE: Biomarkers in Cancer Medicine improving Health Care, Education and Innovation and Lunds universitet
Nature, ISSN 0028-0836, 2014, Volume 511, Issue 7510, pp. 428 - 434
Journal Article