Search Results - "de Moerloose, Barbara"
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Long non-coding RNAs as novel therapeutic targets in juvenile myelomonocytic leukemia
Published in Scientific reports (02-02-2021)“…Juvenile myelomonocytic leukemia (JMML) treatment primarily relies on hematopoietic stem cell transplantation and results in long-term overall survival of…”
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Publisher Correction: Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes
Published in Nature medicine (01-12-2021)Get full text
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Deciphering the Non-Coding RNA Landscape of Pediatric Acute Myeloid Leukemia
Published in Cancers (22-04-2022)“…Pediatric acute myeloid leukemia (pedAML) is a heterogeneous blood cancer that affects children. Although survival rates have significantly improved over the…”
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PATHWAY-DIRECTED THERAPY APPROACH IN PEDIATRIC MDS AND AML
Published in EJC paediatric oncology (01-12-2023)Get full text
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Genetic mechanisms of target antigen loss in CAR19 therapy of acute lymphoblastic leukemia
Published in Nature medicine (01-10-2018)“…We identified genetic mutations in CD19 and loss of heterozygosity at the time of CD19 – relapse to chimeric antigen receptor (CAR) therapy. The mutations are…”
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Prevalence, clinical characteristics, and prognosis of GATA2-related myelodysplastic syndromes in children and adolescents
Published in Blood (17-03-2016)“…Germline GATA2 mutations cause cellular deficiencies with high propensity for myeloid disease. We investigated 426 children and adolescents with primary…”
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Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes
Published in Nature medicine (01-10-2021)“…Germline SAMD9 and SAMD9L mutations ( SAMD9/9L mut ) predispose to myelodysplastic syndromes (MDS) with propensity for somatic rescue. In this study, we…”
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Collaborative Efforts Driving Progress in Pediatric Acute Myeloid Leukemia
Published in Journal of clinical oncology (20-09-2015)“…Diagnosis, treatment, response monitoring, and outcome of pediatric acute myeloid leukemia (AML) have made enormous progress during the past decades. Because…”
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A Novel l-Asparaginase with low l-Glutaminase Coactivity Is Highly Efficacious against Both T- and B-cell Acute Lymphoblastic Leukemias In Vivo
Published in Cancer research (Chicago, Ill.) (15-03-2018)“…Acute lymphoblastic leukemia (ALL) is the most common type of pediatric cancer, although about 4 of every 10 cases occur in adults. The enzyme drug…”
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RAS-pathway mutation patterns define epigenetic subclasses in juvenile myelomonocytic leukemia
Published in Nature communications (19-12-2017)“…Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative disorder of early childhood characterized by mutations activating RAS signaling…”
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TARP as antigen in cancer immunotherapy
Published in Cancer Immunology, Immunotherapy (01-11-2021)“…In recent decades, immunotherapy has become a pivotal element in cancer treatment. A remaining challenge is the identification of cancer-associated antigens…”
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Characterization of Pediatric Acute Myeloid Leukemia With t(7;12)(q36;p13)
Published in Genes chromosomes & cancer (01-11-2024)“…ABSTRACT Acute myeloid leukemia (AML) with t(7;12)(q36;p13) is a recurrent translocation in AML in infants or very young children and was recently included in…”
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Hematopoietic stem cell transplantation in children and adolescents with GATA2-related myelodysplastic syndrome
Published in Bone marrow transplantation (Basingstoke) (01-11-2021)“…GATA2 deficiency is a heterogeneous multi-system disorder characterized by a high risk of developing myelodysplastic syndrome (MDS) and myeloid leukemia. We…”
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Synonymous GATA2 mutations result in selective loss of mutated RNA and are common in patients with GATA2 deficiency
Published in Leukemia (01-10-2020)“…Deficiency of the transcription factor GATA2 is a highly penetrant genetic disorder predisposing to myelodysplastic syndromes (MDS) and immunodeficiency. It…”
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In vivo stabilization of a less toxic asparaginase variant leads to a durable antitumor response in acute leukemia
Published in Haematologica (Roma) (01-02-2023)“…Asparagine is a non-essential amino acid since it can either be taken up via the diet or synthesized by asparagine synthetase. Acute lymphoblastic leukemia…”
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Prognostic discrimination based on the EUTOS long-term survival score within the International Registry for Chronic Myeloid Leukemia in children and adolescents
Published in Haematologica (Roma) (01-10-2017)“…The EUTOS Long-Term Survival score was tested in 350 children with chronic myeloid leukemia in first chronic phase treated with imatinib and registered in the…”
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Characteristics and outcome of primary resistant disease in paediatric acute myeloid leukaemia
Published in British journal of haematology (01-05-2023)“…Summary A significant proportion of events in paediatric acute myeloid leukaemia (AML) are caused by resistant disease (RD). We investigated clinical and…”
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Deciphering molecular heterogeneity in pediatric AML using a cancer vs. normal transcriptomic approach
Published in Pediatric research (01-05-2021)“…Background Still 30–40% of pediatric acute myeloid leukemia (pedAML) patients relapse. Delineation of the transcriptomic profile of leukemic subpopulations…”
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Heterogeneous cytogenetic subgroups and outcomes in childhood acute megakaryoblastic leukemia: a retrospective international study
Published in Blood (24-09-2015)“…Comprehensive clinical studies of patients with acute megakaryoblastic leukemia (AMKL) are lacking. We performed an international retrospective study on 490…”
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