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INTRODUCTION ETV6::JAK2 is a fusion known to drive Acute Lymphoblastic Leukaemia (ALL) in the presence of other genomic lesions which define the JAK/STAT class of Philadelphia-like Acute Lymphoblastic Leukaemia (Ph-like ALL). Ph-like ALL comprises approximately 15% of ALL. Patients with mutations or gene fusions signaling through the JAK/STAT pathway have particularly poor prognosis.
View Article and Find Full Text PDFA sort and sequence approach to dissect heterogeneity of response to a self-amplifying RNA vector in a novel human muscle cell line.
Mol Ther Nucleic Acids
March 2025
Department of Infectious Disease, Imperial College London, London W2 1PG, UK.
Self-amplifying RNA (saRNA) is an extremely promising platform because it can produce more protein for less RNA. We used a sort and sequence approach to identify host cell factors associated with transgene expression from saRNA; the hypothesis was that cells with different expression levels would have different transcriptomes. We tested this in CDK4/hTERT immortalized human muscle cells transfected with Venezuelan equine encephalitis virus (VEEV)-derived saRNA encoding GFP.
View Article and Find Full Text PDFAbatacept dose-finding phase II triaL for immune checkpoint inhibitors myocarditis (ACHLYS) trial design.
Arch Cardiovasc Dis
December 2024
Department of pharmacology, Sorbonne Université, Inserm, CIC-1901, AP-HP, Hôpital Pitié-Salpêtrière, 75013 Paris, France.
Background: Immune checkpoint inhibitor (ICI)-induced myocarditis is a life-threatening adverse drug reaction. Abatacept (a CTLA-4-immunoglobulin fusion protein) has been proposed as a compassionate-use treatment for ICI myocarditis (in combination with corticosteroids and ruxolitinib) but no clinical trial has yet been performed. The abatacept dose can be adjusted using real-time assessment of its target, the CD86 receptor occupancy on circulating monocytes (CD86RO).
View Article and Find Full Text PDFThe role of ruxolitinib in the management of acute GVHD.
Transfus Apher Sci
December 2024
University of Health Sciences, Ankara Oncology Training and Research Hospital, Department of Hematology & Apheresis Unit, Ankara, Turkey; Ankara Yildirim Beyazit University, School of Medicine, Department of Internal Medicine, Division of Hematology, Ankara, Turkey.
Background And Objectives: Following an allogeneic hematopoietic stem cell transplant (allo-HSCT), a primary cause of morbidity and mortality is still steroid-refractory acute graft-versus-host disease (SR-aGVHD). Recently, ruxolitinib, an oral inhibitor of JAK1 and JAK2, was approved for use in individuals suffering from SR-aGVHD. This study aimed to analyze the efficacy and toxicity of ruxolitinib in the real world.
View Article and Find Full Text PDFEfficacy of Ruxolitinib in the management of chronic GVHD.
Transfus Apher Sci
December 2024
University of Health Sciences, Ankara Oncology Training and Research Hospital, Department of Hematology & Apheresis Unit, Ankara, Turkey; Ankara Yildirim Beyazit University, School of Medicine, Department of Internal Medicine, Division of Hematology, Ankara, Turkey.
Objectives: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a potentially curative treatment for hematological diseases, with success rates improving due to advancements in conditioning regimens and new anti-graft versus host disease (GVHD) drugs. Ruxolitinib, an oral selective Janus kinase (JAK) 1 and 2 inhibitor has been used to mitigate the effects of various inflammatory and myeloproliferative syndromes, given the JAK kinase pathway's central role in cytokine signaling during inflammatory and immune processes. In this study we aimed to assess ruxolitinib's efficacy in patients with chronic GVHD (cGVHD).
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