Preclinical model development for pilocytic astrocytoma

Autor: F Selt, J Kiss, J Gronych, DTW Jones, T Brummer, AE Kulozik, SM Pfister, T Milde, O Witt

Publikováno v: Klinische Pädiatrie.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::1e2c0263c2c8b9a4d6238efd7530a167
https://doi.org/10.1055/s-0037-1607402

The Corfu delta beta zero thalassemia: a small deletion acts at a distance to selectively abolish beta globin gene expression [published erratum appears in Blood 1988 May;71(5):1509]

Autor: AE Kulozik, N Yarwood, RW Jones

Publikováno v: Blood. 71:457-462

The Corfu delta beta zero thalassemia is characterized by the clinical picture of thalassemia intermedia. In the homozygous state there is a complete absence of hemoglobin (Hb) A and Hb A2 and a high level of Hb F. A DNA fragment containing the gamma

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::26a85438838de21d88d6f750c6a82cca
https://doi.org/10.1182/blood.v71.2.457.bloodjournal712457

Exon Junction Complexes Show a Distributional Bias toward Alternatively Spliced mRNAs and against mRNAs Coding for Ribosomal Proteins

Autor: Hauer C, Sieber J, Schwarzl T, Hollerer I, Curk T, Am, Alleaume, Matthias Hentze, Ae, Kulozik

Publikováno v: Europe PubMed Central

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::88884da78e0f0993d827bca88c267b96
http://europepmc.org/abstract/med/27475226

Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.

Autor: Kwiatkowski JL; Division of Hematology, Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: kwiatkowski@chop.edu., Walters MC; University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA., Hongeng S; Department of Pediatrics, Faculty of Medicine, Ramathibodi Hospital, Mahidol University, Bangkok, Thailand., Yannaki E; Gene and Cell Therapy Center of the Hematology Dept and Hematopoietic Cell Transplantation Unit, George Papanicolaou Hospital, Thessaloniki, Greece., Kulozik AE; Department of Paediatric Oncology, Haematology, Oncology and Immunology, University of Heidelberg, Heidelberg, Germany; Hopp Children's Cancer Centre, Heidelberg, Germany; Molecular Medicine Partnership Unit, European Molecular Biology Laboratory, University of Heidelberg, Heidelberg, Germany; Clinical Co-operation Unit, Paediatric Leukaemia, DKFZ, and Medical Faculty, University of Heidelberg, Heidelberg, Germany., Kunz JB; Department of Paediatric Oncology, Haematology, Oncology and Immunology, University of Heidelberg, Heidelberg, Germany; Hopp Children's Cancer Centre, Heidelberg, Germany., Sauer MG; Department of Pediatric Hematology and Oncology and Blood Stem Cell Transplantation, Hannover Medical School, Hannover, Germany., Thrasher AJ; UCL Great Ormond Street Institute of Child Health, Great Ormond Street Hospital NHS Trust, London, UK., Thuret I; Department of Pediatric Hematology and Oncology, Rare Disease Center for Thalassemia, Hôpital de la Timone, Marseille, France., Lal A; University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA., Tao G; Bluebird Bio, Somerville, MA, USA., Ali S; Bluebird Bio, Somerville, MA, USA., Thakar HL; Bluebird Bio, Somerville, MA, USA., Elliot H; Bluebird Bio, Somerville, MA, USA., Lodaya A; Bluebird Bio, Somerville, MA, USA., Lee J; Bluebird Bio, Somerville, MA, USA., Colvin RA; Bluebird Bio, Somerville, MA, USA., Locatelli F; IRCCS Ospedale Pediatrico Bambino Gesù, Catholic University of the Sacred Heart, Rome, Italy., Thompson AA; Division of Hematology, Children's Hospital of Philadelphia and Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.

Publikováno v: Lancet (London, England) [Lancet] 2024 Nov 30; Vol. 404 (10468), pp. 2175-2186. Date of Electronic Publication: 2024 Nov 08.

Systematic analysis of RNA-binding proteins identifies targetable therapeutic vulnerabilities in osteosarcoma.

Autor: Zhou Y; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany.; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany., Ray PS; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany.; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany., Zhu J; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Stein F; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Rettel M; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Sekaran T; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Sahadevan S; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Perez-Perri JI; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Roth EK; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany.; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany., Myklebost O; Department of Clinical Science, University of Bergen, Bergen, Norway.; Department of Tumor Biology, Institute for Cancer Research, Oslo University Hospital, Oslo, Norway., Meza-Zepeda LA; Genomics Core Facility, Department of Core Facilities, Institute for Cancer Research, Oslo University Hospital, Oslo, Norway., von Deimling A; Department of Neuropathology, Institute of Pathology, Heidelberg University Hospital, Heidelberg, Germany.; Clinical Cooperation Unit Neuropathology, German Cancer Research Center (DKFZ), German Cancer Consortium (DKTK), and Hopp Children's Cancer Center at the NCT Heidelberg (KiTZ), Heidelberg, Germany., Fu C; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany., Brosig AN; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany.; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany.; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany., Boye K; Department of Oncology, Oslo University Hospital, Oslo, Norway., Nathrath M; Department of Pediatrics and Children's Cancer Research Center, Technical University of Munich, School of Medicine, Munich, Germany.; Pediatric Hematology and Oncology, Klinikum Kassel, Kassel, Germany.; Department of Pediatric Oncology, Hematology and Immunology, Olga Hospital, Stuttgart, Germany., Blattmann C; Department of Pediatric Oncology, Hematology and Immunology, Olga Hospital, Stuttgart, Germany., Lehner B; Department of Orthopaedics, Trauma Surgery and Paraplegiology, Heidelberg University Hospital, Heidelberg, Germany., Hentze MW; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany. hentze@embl.org.; European Molecular Biology Laboratory (EMBL), Heidelberg, Germany. hentze@embl.org., Kulozik AE; Molecular Medicine Partnership Unit (MMPU), Heidelberg University and European Molecular Biology Laboratory (EMBL), Heidelberg, Germany. andreas.kulozik@med.uni-heidelberg.de.; Department of Pediatric Oncology, Hematology and Immunology, Heidelberg University Hospital, Heidelberg, Germany. andreas.kulozik@med.uni-heidelberg.de.; Clinical Cooperation Unit Pediatric Leukemia, German Cancer Research Center (DKFZ) and Heidelberg University, Heidelberg, Germany. andreas.kulozik@med.uni-heidelberg.de.

Publikováno v: Nature communications [Nat Commun] 2024 Apr 01; Vol. 15 (1), pp. 2810. Date of Electronic Publication: 2024 Apr 01.