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Journal articles on the topic 'CRISPR/Cas9, flow cytometry, order of event'

Author: Grafiati

Published: 14 March 2023

Last updated: 31 July 2025

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1

Adriaanse, Fabienne R. S., Sadie M. Sakurada, Shondra M. Pruett-Miller, Ronald W. Stam, Michel C. Zwaan, and Tanja A. Gruber. "Non-Coding HOX Fusions in Pediatric Non-Down Syndrome Acute Megakaryoblastic Leukemia." Blood 134, Supplement_1 (2019): 533. http://dx.doi.org/10.1182/blood-2019-127014.

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The homeobox (HOX) genes are a highly conserved family of transcription factors involved in embryonic patterning as well as adult hematopoiesis. Dysregulation of HOX genes, in particular upregulation of HOXA cluster genes, is a frequent event in Acute Myelogenous Leukemia (AML). Recently, we performed a detailed genomic analysis on pediatric non-Down Syndrome Acute Megakaryoblastic Leukemia (non-DS-AMKL) and identified novel fusions involving a HOX cluster gene in 14.9% of the cases. While most fusions were predicted to lead to an in-frame functional protein, several fusions included a non-cod

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Ivy, Kathryn S., Candace H. Cote, and Paul Brent Ferrell. "IDH2 Mutations Induce Altered STAT Signaling and Cytokine Responses Which Are Restored By Enasidenib." Blood 132, Supplement 1 (2018): 1468. http://dx.doi.org/10.1182/blood-2018-99-117783.

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Abstract Acute myeloid leukemia (AML) is a heterogeneous myeloid malignancy characterized by mutational and clonal heterogeneity. Mutations in isocitrate dehydrogenase 1 and 2 (IDH1/2) are common, occurring in approximately 15-20% of patients, and actionable, with recently approved inhibitors for both mutations. These inhibitors lead to leukemia cell differentiation in vitro, in vivo and in patients. Healthy myeloid differentiation is governed by precise regulation of intracellular signaling, but this regulation is disrupted in AML. Given that signal transducer and activator of transcription (

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Pabst, Gabriel, Johannes Foßelteder, Angelika Schlacher, et al. "Modeling the Development of SRSF2 Mutated Myeloid Malignancies By CRISPR/Cas9 Mediated Genome Engineering of Primary Human Hematopoietic Stem and Progenitor Cells." Blood 138, Supplement 1 (2021): 2160. http://dx.doi.org/10.1182/blood-2021-149591.

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Abstract Introduction: Acute Myeloid Leukemia (AML) is a malignant disease of the bone marrow that can arise from a premalignant condition called clonal hematopoiesis of indeterminate potential (CHIP). Mutations in Serine and Arginine-rich Splicing Factor 2 (SRSF2) are detected in CHIP and mediate a high risk for AML development. Here we used CRISPR/Cas9-mediated genome engineering to introduce a heterozygous SRSF2P95H mutation into primary human hematopoietic stem and progenitor cells (HSPCs) and investigated its functional consequences using both in vitro and in vivo assays. Methods: We used

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Xiong, Kun. "Feasibility assessment of using cRISPR-Cas9 to improve the infiltration of CAR-T cells in solid tumors." Theoretical and Natural Science 60, no. 1 (2024): 46–51. http://dx.doi.org/10.54254/2753-8818/60/20241395.

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Abstract. Chimeric antigen receptor T-cell immunotherapy (CAR-T) has been developing for decades, CAR-T is playing an increasingly important role in tumor treatment. However, because fibroblasts (CAFs) in solid tumors secrete proteins and glycans to form ECM, CAR-T is faced with the challenge of improving tumor invasion. To this end, a new scheme was put forth to alter CAR T cells such that they release heparinase (HPSE) to break down heparan sulfate proteoglycan (HSPG), which is covered on the outermost layer of the cancerous cells by CAF and released. In order to solve the above problems, CR

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Lanjewar, S. N., and K. R. Bondioli. "205 Optimization of Transfection Efficiency for CRISPR/Cas9-Induced Genomic Editing in Porcine Fibroblast Cells." Reproduction, Fertility and Development 30, no. 1 (2018): 243. http://dx.doi.org/10.1071/rdv30n1ab205.

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The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas9) system creates DNA double-stranded breaks (DSB) at specific sequences and allows efficient genomic modification, even in species previously resistant to gene editing. The DSB can be repaired using non-homologous end joining (creating insertions/deletions) or by homology directed repair (HDR) using a donor DNA with small changes at the cut site, giving rise to precise targeted modifications. Despite growing interest in genome editing using RNA-guided endonucleases, the efficiency of HDR is only 0.5 t

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Haase-Kohn, Cathleen, Markus Laube, Cornelius K. Donat, Birgit Belter, and Jens Pietzsch. "CRISPR/Cas9 Mediated Knockout of Cyclooxygenase-2 Gene Inhibits Invasiveness in A2058 Melanoma Cells." Cells 11, no. 4 (2022): 749. http://dx.doi.org/10.3390/cells11040749.

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The inducible isoenzyme cyclooxygenase-2 (COX-2) is an important hub in cellular signaling, which contributes to tumor progression by modulating and enhancing a pro-inflammatory tumor microenvironment, tumor growth, apoptosis resistance, angiogenesis and metastasis. In order to understand the role of COX-2 expression in melanoma, we investigated the functional knockout effect of COX-2 in A2058 human melanoma cells. COX-2 knockout was validated by Western blot and flow cytometry analysis. When comparing COX-2 knockout cells to controls, we observed significantly reduced invasion, colony and sph

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Beigl, Tobias B., Ine Kjosås, Emilie Seljeseth, Nina Glomnes, and Henriette Aksnes. "Efficient and crucial quality control of HAP1 cell ploidy status." Biology Open 9, no. 11 (2020): bio057174. http://dx.doi.org/10.1242/bio.057174.

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ABSTRACTThe near-haploid human cell line HAP1 recently became a popular subject for CRISPR/Cas9 editing, since only one allele requires modification. Through the gene-editing service at Horizon Discovery, there are at present more than 7500 edited cell lines available and the number continuously increases. The haploid nature of HAP1 is unstable as cultures become diploid with time. Here, we demonstrated some fundamental differences between haploid and diploid HAP1 cells, hence underlining the need for taking control over ploidy status in HAP1 cultures prior to phenotyping. Consequently, we opt

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Foßelteder, Johannes, Angelika Schlacher, Gabriel Pabst, et al. "Introduction and Genetic Correction of Calreticulin Mutations in Human Hematopoietic Stem and Progenitor Cells Sheds Light on MPN Pathogenesis." Blood 138, Supplement 1 (2021): 2541. http://dx.doi.org/10.1182/blood-2021-147919.

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Abstract Introduction: Recurrent mutations in calreticulin (CALR) are present in 70% to 80% of essential thrombocythemia (ET) and primary myelofibrosis (PMF) patients without a JAK2 or MPL mutation. Despite recent advances in understanding mutant CALR, the detailed mechanisms are not fully elucidated, and current knowledge is mainly based on transgenic mouse models or human cancer cell lines. Thus, to more faithfully model MPN pathogenesis, we first aimed to introduce heterozygous type-1 and type-2 CALR mutations into healthy human hematopoietic stem and progenitor cells (HSPCs) via targeted C

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Castro, Jesus, Mariana Gaelzer, and Scott Welford. "Abstract 209: Elucidating the role of NMDA subunit NR2B in non-tumor and tumor-bearing mice." Cancer Research 82, no. 12_Supplement (2022): 209. http://dx.doi.org/10.1158/1538-7445.am2022-209.

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Abstract Therapeutic options for glioblastoma multiforme (GBM), the most common and fatal brain tumor, have been limited over the course of recent decades, leaving radiation as one of the few effective therapies. While advancements in radiation therapy have improved life expectancy and patient outcome, exposure to radiotherapy causes normal tissue toxicity and damage to the central nervous system, resulting in cognitive impairment and negative patient side effects. In the hippocampus, the memory processing center of the brain, radiotherapy-induced elevated glutamate levels lead to over-excitat

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Soerensen, Johannes Frasez, Carina Agerbo Rosenberg, Katharina Wolter, et al. "Assessing Lipid Nanoparticle RNA Delivery Including CRISPR-Cas9 Based Therapy to Bone Marrow Cells with Emphasis on Leukemic Blasts - a Proof-of-Principle Study." Blood 144, Supplement 1 (2024): 7451. https://doi.org/10.1182/blood-2024-199992.

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Acute myeloid leukemia (AML) with the translocation t(8;21)(q22;q22.1) resulting in the fusion oncogene RUNX1::RUNX1T1 is a well-described subtype of AML. Generally perceived as associated with a favorable prognosis, the main cause of mortality in these patients remain relapse, occurring in an estimated 40% of patients leading to increased mortality. Gene editing technology CRISPR-Cas9, has in previous research been demonstrated to be able to disrupt the RUNX1::RUNX1T1 fusion gene. The disruption leads to inhibited leukemic cell growth and proliferation, suggesting its potential as a future th

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Kaur, Surinder, Stephen C. Alley, Matt Szapacs, et al. "2021 White Paper on Recent Issues in Bioanalysis: Mass Spec of Proteins, Extracellular Vesicles, CRISPR, Chiral Assays, Oligos; Nanomedicines Bioanalysis; ICH M10 Section 7.1; Non-Liquid & Rare Matrices; Regulatory Inputs (Part 1A – Recommendations on Endogenous Compounds, Small Molecules, Complex Methods, Regulated Mass Spec of Large Molecules, Small Molecule, PoC & Part 1B - Regulatory Agencies' Inputs on Bioanalysis, Biomarkers, Immunogenicity, Gene & Cell Therapy and Vaccine)." Bioanalysis 14, no. 9 (2022): 505–80. http://dx.doi.org/10.4155/bio-2022-0078.

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The 15th edition of the Workshop on Recent Issues in Bioanalysis (15th WRIB) was held on 27 September to 1 October 2021. Even with a last-minute move from in-person to virtual, an overwhelmingly high number of nearly 900 professionals representing pharma and biotech companies, contract research organizations (CROs), and multiple regulatory agencies still eagerly convened to actively discuss the most current topics of interest in bioanalysis. The 15th WRIB included 3 Main Workshops and 7 Specialized Workshops that together spanned 1 week in order to allow exhaustive and thorough coverage of all

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Putino, Rossana, Alessia Boaretto, Gennaro Bruno та ін. "Abstract B110: β3-adrenergic receptor as a new molecular target in neuroblastoma treatment". Molecular Cancer Therapeutics 22, № 12_Supplement (2023): B110. http://dx.doi.org/10.1158/1535-7163.targ-23-b110.

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Abstract Purpose of study: β3-Adrenergic receptor correlation with tumor growth and progression in preclinical and clinical studies in neuroblastoma. Experimental procedures: Knock-out of β3-Adrenergic Receptor (β3-AR) in Neuroblastoma (NB) cell lines using CRISPR-Cas9 technology. In vivo experiment using β3-AR knock-out cells in order to evaluate the tumor growth in mice. RNA Sequencing of tumor masses derived from mice to investigate β3-AR Dependent tumor signaling pathways. Flow Cytometry analysis to observe the expression level of β3- Adrenergic Receptor on circulating tumor cells (CTCs) i

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Eken, Janneke, Fleur R. M. Havenaar, Edwin Quinten, et al. "Oncogenic CARD11 Mutations and Autonomous BCR Signaling Act As Functionally Equivalent Alternative Drivers in ABC-DLBCL." Blood 144, Supplement 1 (2024): 1619. https://doi.org/10.1182/blood-2024-204704.

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Diffuse large B-cell lymphoma of activated B-cell type (ABC-DLBCL) is characterized by chronic signaling of the B-cell receptor (BCR) pathway with constitutive NF-kB activation. We have recently identified autonomous, i.e. antigen-independent, signaling originating from individual BCR complexes as the cause of BCR pathway activation in the majority of ABC-DLBCL cases (Eken et al., J Exp Med 2024). As a pure immunological driver, autonomous BCR signaling cannot be recognized by sequence analysis nor be readily added to proposed genetic DLBCL subclassifications. Besides autonomous BCR signaling,

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Qazi, Areebah, Sophia Agrusa, Swornalata Pukhrambam, et al. "Abstract 1455: Impact of ZFHX3 loss on endometrial cancer progression." Cancer Research 85, no. 8_Supplement_1 (2025): 1455. https://doi.org/10.1158/1538-7445.am2025-1455.

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Abstract Endometrial cancer (EC) is the sixth most common cancer among women and ranks as the leading type of gynecological cancer. Although EC is often diagnosed early, current treatment plans significantly limit fertility-sparing options, posing challenges for younger patients. This research focuses on the role of the gene ZFHX3 (ATBF1) to understand its effects on the advancement of endometrial cancer. In order to investigate this, we first transfected 12Z-ESR1 epithelial endometrial cells with ZFHX3 siRNA to create transient gene knockdowns. Following cell collection, western blotting was

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Ranjan, Nikhil, Michael Cole, Gloria F. Gerber, et al. "Genetic or Epigenetic CR1 Deficiency Defines Catastrophic Antiphospholipid Syndrome (CAPS) and Response to Complement Inhibition." Blood 144, Supplement 1 (2024): 138. https://doi.org/10.1182/blood-2024-207793.

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Introduction: CAPS is a life-threatening manifestation of the antiphospholipid syndrome (APS) characterized by widespread thrombosis and multi-organ failure. Despite standard-of-care therapies, CAPS is associated with &gt;30% mortality. Complement is implicated in the pathophysiology of thrombosis in CAPS, and complement inhibition shows efficacy as salvage therapy. We previously used the modified HAM (mHam) assay to demonstrate complement activation in &gt;85% of CAPS sera (Chaturvedi, Blood, 2020). Further, 48% (9/19) of CAPS patients had rare germline variants in complement regulato

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Dong, Mengmeng, Enfan Zhang, Haimeng Yan, et al. "Macrophages Promote DNA Repair of Double Strand Break in Multiple Myeloma Cells By Non-Homologous End Joining(NHEJ), Nevertheless Decrease Its Accuracy." Blood 134, Supplement_1 (2019): 3087. http://dx.doi.org/10.1182/blood-2019-126753.

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Multiple myeloma (MM) is a hematological malignancy of B cells, characterized by clonal proliferation of malignant plasma cells. DNA damage and genomic instability play an important role in the pathogenesis of MM. Based on the characteristics of high heterogeneity and genomic instability of MM, and the protective effect of MΦs on MM cells (MMCs), our study intended to further clarify whether MΦs affect MMCs DNA damage response (DDR) and DNA repair, and the relationship between MΦs and genomic instability of MMCs. We found that the content of MΦs in bone marrow biopsy of MM patients was related

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Corat, Marcus A. F., Jean-Yves Metais, and Cynthia E. Dunbar. "Progress Towards Creation of a Rhesus Macaque Animal Model for PNH Disease Via Crispr/Cas9 Technology to Knock out the PIG-a Gene." Blood 124, no. 21 (2014): 4389. http://dx.doi.org/10.1182/blood.v124.21.4389.4389.

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Abstract Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic stem/progenitor cell (HSPC) disease characterized by severe intravascular hemolysis, bone marrow failure, and propensity to thrombosis, causing early death in untreated patients. PNH has been linked to acquired somatic loss-of-function mutations in the X-linked PIG-A gene in HSPC, with resultant disruption of the first step of the biosynthesis of GPI anchors and loss of cell-surface expression of GPI-linked proteins such as CD55 or CD59. PNH red cells are sensitive to complement-mediated lysis due to loss of

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Chen, Manling, Yihan Mei, Xiaoyu Liu, et al. "Pre-Leukemic Transformation of the Bone Marrow Microenvironment Induced By AML1-ETO Fusion Protein." Blood 142, Supplement 1 (2023): 5613. http://dx.doi.org/10.1182/blood-2023-179881.

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Objective: The bone marrow (BM) microenvironment, especially stromal cells (SCs) and cytokines, plays a critical role in supporting normal hematopoiesis. However, in cases of hematological malignancies such as acute myeloid leukemia (AML), leukemia cells can reshape the BM microenvironment to promote their survival. Despite this, there is still limited understanding of the changes in AML1-ETO fusion protein induced pre-leukemia microenvironment niche. In this study, the alterations of the BM microenvironment in the Aml1 Eto/+; Mx1-Cre (AE KI) mouse model are thoroughly explored and analyzed. T

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Wakabayashi, Aoi, Maryanne Kihiu, Malini Sharma, et al. "Interrogating Post-Transcriptional Mechanisms of Fetal Hemoglobin Regulation." Blood 138, Supplement 1 (2021): 3079. http://dx.doi.org/10.1182/blood-2021-151488.

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Abstract Elevated levels of fetal hemoglobin (HbF) significantly ameliorate clinical outcomes for patients with beta-hemoglobinopathies, such as sickle cell disease (SCD). The only FDA-approved drug for treating SCD through inducing HbF is hydroxyurea, however the mechanism of action is unknown with variable effectiveness among patients. Thus, there remains a strong interest to identify more robust means of upregulating HbF, such as specific inhibition of HbF repressors. BCL11A and LRF are well-characterized transcription factors that independently repress the fetal type b-globin like genes HB

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Barta, Stefan K., Marco Ruella, Stephen J. Schuster, et al. "CD5-Deleted Chimeric Antigen Receptor Cells (Senza5™ CART5) to Enhance Immunotherapy Against T-Cell Non-Hodgkin Lymphoma: A First-in-Human Phase I Clinical Trial." Blood 144, Supplement 1 (2024): 7221. https://doi.org/10.1182/blood-2024-201063.

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Background and Significance: Outcomes for relapsed or refractory (RR) T-cell lymphomas (TCL) are poor with standard therapies and cure is elusive. While in B-cell malignancies CAR T-cell (CART) therapy has changed the treatment paradigm, its development in TCL faces many challenges, mainly related to the fact that normal and malignant T cells share similar antigens. This can result in fratricide during CART manufacture and T-cell aplasia after CART treatment. To overcome these obstacles, we used CRISPR-Cas9 CD5 short-guide RNA to delete CD5 (Senza5™) in T cells and created a 4-1BB costimulated

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Berrien-Elliott, Melissa M., Wong Pamela, Carly Neal, et al. "Primary Human NK Cell Gene-Editing Reveals a Critical Role for NKG2A in Cytokine-Induced Memory-like NK Cell Responses." Blood 134, Supplement_1 (2019): 3237. http://dx.doi.org/10.1182/blood-2019-129162.

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Natural killer (NK) cells are an emerging cellular immunotherapy for patients with acute myeloid leukemia (AML); however, the best approach to maximize NK cell anti-leukemia potential is unclear. Paradigm-shifting reports have shown that NK cells exhibit "memory-like" properties following hapten exposure, virus infection, or combined cytokine pre-activation. Human cytokine-induced memory-like (ML) NK cells display enhanced re-stimulation responses to numerous activating stimuli, including tumor target cells. This has been translated in clinical trials as cellular therapy for rel/ref AML patien

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Zhang, Yanlei, Hechun Ma, Boang Han, et al. "Abstract 2666: Genetic ablation of B2M leads to resistance to PD-1/PD-L1 blockade in vivo." Cancer Research 84, no. 6_Supplement (2024): 2666. http://dx.doi.org/10.1158/1538-7445.am2024-2666.

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Abstract Immune checkpoint inhibitors (ICIs) has revolutionized the treatment landscape of various cancers by reinvigorating the exhausted T cells in patients. However, the therapeutic efficacy is largely confined due to the primary or acquired resistance to anti-PD-1 (L1) therapy in many patients. Recently, the mechanisms of resistance to ICIs has been extensively elucidated from different aspects and emerging sequencing data from clinical samples has pointed to IFN-γ signaling defects and antigen presentation loss in patients who are resistant to PD-1(L1) blockade. The loss-of-function mutat

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Pilunov, Artem, Dmitrii Romaniuk, Savely Sheetikov, et al. "Development of T-Cell Therapy Targeting Hematopoietic Minor Histocompatibility Antigen HA-1." Blood 134, Supplement_1 (2019): 5749. http://dx.doi.org/10.1182/blood-2019-130552.

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Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is currently the only curative therapy for hematological malignancies yet in nearly one-third of patients, it is followed by a relapse of the disease contributing to high mortality. In fully HLA-matched allo-HSCT graft versus leukemia reaction is driven by the recognition of the minor histocompatibility antigens (MiHAs) - endogenous polymorphic peptides presented by MHC. Particularly, HA-1 MiHA is a promising target for immunotherapy. HA-1 is presented by frequent among Caucasians HLA allele - A*02:01. The single nucleotide variati

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Shen, Yandong, Kyle R. Crassini, Michael E. O'Dwyer, et al. "The Dual PI3/PIM-Kinase Inhibitor, Ibl-202, Is Highly Synergistic with Venetoclax Against CLL Cells, and TP53-Knock-out Cells, and Under Conditions That Mimic the Tumor Microenvironment." Blood 132, Supplement 1 (2018): 1870. http://dx.doi.org/10.1182/blood-2018-99-115574.

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Abstract Background The B-cell receptor (BCR) signaling pathway and the pro-survival Bcl-2-family of proteins play crucial roles in the pathogenesis of chronic lymphocytic leukemia (CLL). Constitutive activity of the BCR signaling pathway and overexpression of Bcl-2 promote CLL-cell survival, proliferation and drug resistance. BCR-targeted therapies, most notably ibrutinib and idelalisib and the Bcl-2 inhibitor Venetoclax, demonstrate the potential of targeting these pathways. However, there is no evidence that these novel agents are curative in the event of relapse. Treatment options remain l

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Gupta, Dipti, Yannis Hara, Samuel Lessard, et al. "Catalytic Activity of Heme-Regulated eIF2 Alpha Kinase (HRI) Regulates Fetal Hemoglobin." Blood 136, Supplement 1 (2020): 7. http://dx.doi.org/10.1182/blood-2020-139991.

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Sickle cell disease (SCD) is a hereditary disorder occurring due to a mutation in the β- globin gene resulting in hemoglobin polymerization and sickling of red blood cells that drives an array of severe pathophysiologies. SCD patients with hereditary persistence of fetal hemoglobin mutations show amelioration of disease symptoms. HRI is a heme sensing eIF2α kinase belonging to the integrated stress response pathway, primarily regulating the hemoglobin synthesis in RBCs. Under low levels of heme, HRI undergoes auto-phosphorylation and subsequently phosphorylates its substrate eIF2α. This impede

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Iyer, Prajish, Brian Jiang, Girish Venkataraman, et al. "Glycerolipid Metabolism Via the MGA-MYC-NME1-PGP Axis As a Key Regulator of Oxidative Phosphorylation in Richter's Transformation." Blood 142, Supplement 1 (2023): 79. http://dx.doi.org/10.1182/blood-2023-179427.

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Despite advances in targeted therapies for chronic lymphocytic leukemia (CLL), Richter's transformation (RT) remains a clinical challenge. RT is an aggressive shift from CLL to lymphoma, associated with metabolic changes and aberrations in the MYC transcriptional network. Genomic analysis of paired CLL and RT cases revealed recurrent mutations or deletions in the MYC suppressor Max-gene-associated (MGA) increase from 5% (CLL) to 30% (RT) cases, highlighting its critical yet unexplored role as a driver event. We recently developed a murine RT model using B cell-restricted in vitro editing of LS

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Wenthe, Sophia, Kelsie Becklin, Brett Napiwocki, Emma Kozurek, Branden Moriarity, and Jong Hyuk Kim. "Abstract 198: Unveiling chromatin accessibility landscape and convergent oncogenic pathway in angiosarcoma models using induced pluripotent stem cells." Cancer Research 82, no. 12_Supplement (2022): 198. http://dx.doi.org/10.1158/1538-7445.am2022-198.

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Abstract Angiosarcoma is a rare soft tissue sarcoma that forms malignant vessels. Angiosarcomas are aggressive and highly metastatic, resulting in a poor prognosis. Recurrent somatic mutations in TP53 and genes involved in PI3K/AKT/mTOR pathway such as PIK3CA and PIK3R1 are identified in angiosarcomas. However, angiosarcomas are genomically complex, and the oncogenic mechanisms are virtually unknown. Due to its rarity, establishment of experimental tumor models is an unmet need for angiosarcoma research. In this study, we used human induced pluripotent stem cells (iPSCs) to develop a novel, re

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Shi, Yuanfei, Yanchun Zhao, Yi Xu, et al. "Effects and Molecular Mechanism of Inhibiting p53 Signaling Pathway By NSUN6 on the Resistance to BCL-2 Inhibitor for Diffuse Large B-Cell Lymphoma." Blood 144, Supplement 1 (2024): 6238. https://doi.org/10.1182/blood-2024-202205.

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Background: Most DLBCL patients with recurrent and refractory DLBCL have high BCL-2 expression, but some patients are resistant to BCL-2 inhibitors for unknown reasons, the mechanism of which is unknown yet. We constructed a DLBCL cell line resistant to venetoclax in the early stage to explore its molecular mechanism of resistance. We found that m5C methyltransferase NSUN6 was highly expressed in DLBCL resistant cell lines, and also highly expressed in lymph nodes of relapsed and refractory DLBCL patients. Those with high expression had poor prognosis. Knocking down NSUN6 can significantly red

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McCONVILLE, Michael, Toby Thomas, Catherine Valadez, et al. "Familial Thrombocytopenia-Associated Germline ETV6 P214L Mutation Results in XPO1-Mediated Nuclear Export." Blood 142, Supplement 1 (2023): 1298. http://dx.doi.org/10.1182/blood-2023-182269.

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In recent years, a number of heterozygous germline mutations in ETV6 have been associated with familial thrombocytopenia and predisposition to hematologic malignancies. While most of these missense mutations occur in the DNA-binding-domain of ETV6, the recurrent P214L mutation is unique in that it occurs in the central disordered region of the protein. The ETV6 P214L mutation has previously been shown to disrupt the nuclear localization of ETV6 and the ability of ETV6 to act as a transcriptional repressor. Unexpectedly, we have found that the P214L missense mutation creates a recognition motif

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Liu, Jile, Xiaomei Zhang, Rui Sun, et al. "Oncolytic Herpes Simplex Virus-Loaded CD19 CAR-T Cells Improves the Therapeutic Effect of Diffuse Large B-Cell Lymphoma." Blood 144, Supplement 1 (2024): 4810. https://doi.org/10.1182/blood-2024-205138.

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Background In the clinical practice of treating patients with diffuse large B-cell lymphoma, CD19 CAR-T cell therapy has failed to achieve the desired therapeutic effect and is highly prone to recurrence. In the field of solid tumors, the combined therapy of CAR-T cell therapy and oncolytic viruses has made breakthrough progress. Therefore, we introduce this combined therapeutic idea into the treatment of diffuse large B-cell lymphoma. In order to deal with the characteristic that diffuse large B-cell lymphoma is highly prone to systemic metastasis and to avoid the problem that oncolytic virus

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Walker, Janek S., Kerstin Wenzl, Joseph P. Novak, et al. "Truncating SPEN Mutations Highlight BN2-Subtype DLBCL with Aggressive Biology and Features of Immune Evasion." Blood 142, Supplement 1 (2023): 4140. http://dx.doi.org/10.1182/blood-2023-181974.

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Introduction: Molecular classifications have been developed to characterize the heterogeneity of diffuse large B cell lymphoma (DLBCL), with activation of BCL6 and Notch signaling pathways acting as driving pathogenic features in C1/BN2 subtype tumors. Among C1/BN2 defining variants are truncating/inactivating SPEN mutations. SPEN regulates the Notch signaling pathway by initiating formation of a repressive complex that facilitates transcriptional repression of Notch target genes. Our group has identified SPEN mutations enriched in DLBCL cases failing to achieve event-free status at 24 months

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Skuli, Sarah, A'Ishah Bakayoko, Gerald Wertheim, et al. "The Mevalonate Pathway Is a Therapeutic Target in TP53 Mutant Acute Myeloid Leukemia." Blood 142, Supplement 1 (2023): 408. http://dx.doi.org/10.1182/blood-2023-185059.

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Introduction: Acute myeloid leukemia (AML) with mutations in the TP53 tumor suppressor gene is the most fatal of AMLs with a median overall survival of only six months due to chemotherapy resistance. To understand biologic differences in TP53 mutant (MT) AML, as compared to wildtype (WT) AML, we performed transcriptomic analysis on sorted patient samples. Gene set enrichment analysis demonstrated significant upregulation of the cholesterol biosynthesis or mevalonate pathway in TP53 MT AML. In TP53 MT solid tumor models, the mevalonate pathway plays a key role in tumorigenesis and metastasis. W

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Coutinho de Oliveira, Beatriz, Saaurav Bari, Jessica Kerr, et al. "Abstract 3807: Validation of a functional genomics screen based on lentiviral integration site analysis reveals single-gene disruptions enhancing CAR19 T-cell effectiveness in preclinical models." Cancer Research 85, no. 8_Supplement_1 (2025): 3807. https://doi.org/10.1158/1538-7445.am2025-3807.

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Abstract Anti-CD19 CAR (CAR19) T-cell therapy has revolutionized the treatment for relapsed and refractory B-cell malignancies, showing long-term efficacy and potential cures. However, while many patients, including those with Chronic Lymphocytic Leukemia (CLL), exhibit initial signs of response, nearly two-thirds will eventually relapse. This highlights the critical need to understand treatment failure mechanisms and to identify targets to improve therapies. Our team previously reported an exceptional response in a CLL patient treated with CAR19 T-cells, which coincided with significant expan

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Isabelle, Colleen, Amy Boles, Kathleen McConnell, et al. "Pathobiology and Targeting of CD38 in Cutaneous T-Cell Lymphoma." Blood 142, Supplement 1 (2023): 1650. http://dx.doi.org/10.1182/blood-2023-189556.

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Introduction: Cutaneous T-cell lymphoma (CTCL) is a malignancy of mature CD4+ T-cells that primarily affects the skin. Despite a wide variety of topical and systemic therapies for patients, CTCL remains difficult to treat. Because of its high likelihood of relapse and resistance, new therapeutic approaches are needed. We recently showed strong CD38 expression on neoplastic T-cells from patients with mature T-cell neoplasms, including CTCL. In this study, we evaluated i) the role of CD38 in CTCL pathogenesis ii) strategies to enhance CD38 expression in CTCL and iii) tested efficacy of combinati

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Aruljothi, Charlesantony, Subin S. George, Patrick Somers, et al. "Regulation of Ribosomal RNA Synthesis in Myeloid Progenitors By Cell Type Specific Transcription Factors." Blood 136, Supplement 1 (2020): 11–12. http://dx.doi.org/10.1182/blood-2020-138776.

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Haematopoiesis relies on the ability of hematopoietic stem cells to progress through a systematic hierarchy to produce lineage-restricted progenitors that terminally differentiate into phenotypically distinct types of mature hematopoietic cells. This process is precisely coordinated by the combinatorial activity of lineage-specifying transcription factors (TFs). Indeed, the critical transcriptional program of every hematopoietic cell type, and indeed of all cell types throughout the body, requires a set of core TFs for its proper execution. A frequently-overlooked component of the cellular tra

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Barwe, Sonali P., Meredith Tavenner, Patrick Van Berkel, et al. "Identification of DLK1 As a Novel Therapeutic Target in Down Syndrome Myeloid Leukemia." Blood 144, Supplement 1 (2024): 209. https://doi.org/10.1182/blood-2024-211364.

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The current standard-of-care for myeloid leukemia associated with Down syndrome (ML-DS) relies on cytotoxic chemotherapy. Although primary ML-DS has favorable prognosis, the 10-20% of patients who develop relapsed leukemia or have refractory disease show a 3-year event-free survival less than 21% (Raghuram et al., 2023). Novel treatment options are needed to improve outcome in these patients with relapsed or refractory disease. The genomic landscape of ML-DS has been characterized by multiple studies (Labuhn et al., 2019; Nikolaev et al., 2013; Sato et al., 2024; Yoshida et al., 2013). In addi

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Dahlgren, Anna R., Francesca Careddu, Raymond Adili, et al. "Identification of the Role of SEL1L in Platelet Function with Implications for Atypical Equine Thrombasthenia in Thoroughbred Horses." Blood 142, Supplement 1 (2023): 3944. http://dx.doi.org/10.1182/blood-2023-187146.

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Atypical Equine Thrombasthenia (AET) is a recessive heritable platelet disorder in horses due to aberrant platelet signaling after thrombin stimulation, preventing platelets from fully activating or efficiently binding to fibrinogen, leading to prolonged bleeding. To date, AET has only been identified in the Thoroughbred horse breed. A prevalence study performed at one breeding farm found that one in every 150 Thoroughbreds was affected. Despite the negative effects on horse health and racing performance, the underlying etiology of this unique platelet disorder is unknown. Here we show that a

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Meyer, Tatjana, Nikolaus Jahn, Anna Dolnik, et al. "BRCA1/2 Containing Complex 3 (BRCC36) Is Recurrently Mutated in AML with t(8;21) and Associated with Increased Sensitivity to Chemotherapy through Impairment of the DNA Damage Repair Pathway." Blood 132, Supplement 1 (2018): 1487. http://dx.doi.org/10.1182/blood-2018-99-114143.

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Abstract Introduction BRCA1/BRCA2-containing complex 3 (BRCC36) is a Lys63-specific deubiquitinating enzyme (DUB) involved in DNA damage repair. Mutations in BRCC36 have been identified in 2-3% of patients with myelodysplastic syndromes (MDS) and secondary AML (sAML). The role of BRCC36 mutations in de novo AML and their impact on DNA damage-inducing cytotoxic chemotherapy sensitivity is not clear. Aim We aimed to determine the incidence of BRCC36 mutations in AML and their impact on outcome and drug sensitivity in vitro. Methods We analyzed the entire coding region of BRCC36 for mutations in

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Ghobadi, Armin, Ibrahim Aldoss, Shannon L. Maude, et al. "WU-CART-007 (WT-7), an Allogeneic CAR T-Cell Targeting CD7 in Relapsed/Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/Lymphoma (T-ALL/LBL): Phase 2 Results." Blood 144, Supplement 1 (2024): 3450. https://doi.org/10.1182/blood-2024-202005.

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R/R T-ALL/LBL are challenging malignancies to treat with few treatment options and high rates of relapse and mortality. WT-7 is a CD7-targeted CAR T-cell product with CRISPR/Cas9 deletion of CD7 and T-cell receptor alpha constant (TRAC), to prevent fratricide and enable the use of healthy donor allogeneic T-cells (Leedom, et al. ASH 2021). We report time-to-event (TTE) update from the Phase 2 portion of WU-CART-007 1001 (NCT04984356), a Phase 1/2 study of WU-CART-007 in patients (pts) with R/R T-ALL/LBL, and a focus on subgroup analysis of RP2D. In the Phase 2 portion, pts received a single in

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Stopka, Tomas, Jarmila Vargova, Karina Vargova, et al. "Myristoylated Alanine-Rich C-Kinase Substrate (MARCKS) Is a New Biomarker for Mantle Cell Lymphoma: Expression, Localization, and Phosphorylation Study." Blood 128, no. 22 (2016): 1767. http://dx.doi.org/10.1182/blood.v128.22.1767.1767.

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Abstract Mantle cell lymphoma (MCL) is a relatively distinct B-cell non-Hodgkin lymphoma subtype with aggressive and often recurrent clinical course. At diagnosis, MCL often manifests with leukemization, a feature more common to chronic lymphocytic leukemia (CLL). Common features and differences between MCL and CLL were not yet explored by comprehensive global approaches, despite such understanding potentially being very neat for deciphering pathogenesis and tailoring therapies of these clinically distinct diseases. In our study, we have compared MCL(n=10), CLL(n=10) and normal control(n=8) B-

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Barrio, Santiago, Larissa Haertle, Umair Munawar, et al. "Clonal Competition Models to Understand Progression and Resistance in Myeloma." Blood 134, Supplement_1 (2019): 1807. http://dx.doi.org/10.1182/blood-2019-130812.

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Background: Progression and relapse in Multiple Myeloma (MM) is induced by changes in the clonal tumor composition. In order to better understand the mechanisms underlying these dynamics, we developed clonal competition models based on the co-culture of fluorescent labelled isogenic MM cells, with or without the alteration under study. Methods: To understand the effect of mono- and bi-allelic TP53 lesions, we use the AMO1 cell line, one of few myeloma cell lines harbouring wild type TP53 (WT). After modification with CRISPR / CAS9, we selected subclones with mono- and/or bi-allelic deletion of

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Schimpf, Marlise L. Guerrero, Courtney C. Sparger, Hsuan-Ting Huang, Dean Wade, and Maria E. Figueroa. "Abstract A37: PDZD2 is essential for steady-state hematopoiesis and its 37-kDa secreted product, sPDZD2, functions as a soluble tumor suppressor in AML." Blood Cancer Discovery 4, no. 3_Supplement (2023): A37. http://dx.doi.org/10.1158/2643-3249.aml23-a37.

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Abstract PDZ domain-containing protein 2 (PDZD2) is almost universally silenced through hypermethylation in myeloid malignancies. PDZD2 encodes for a 300kDa protein that it is cleaved into a 37kDa protein that is secreted to the tissue microenvironment (sPDZD2). Given that sPDZD2 has been reported to function as a tumor suppressor in solid tumors and its almost universal loss in AML, we hypothesized that PDZD2 is required for normal hematopoiesis and that sPDZD2 functions as a soluble tumor suppressor in the hematopoietic system. To determine if sPDZD2 is secreted to the bone marrow (BM) micro

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Mishima, Yuji, Jiantao Shi, Michele Moschetta, et al. "In Vivo Analysis of Clonal Evolution of Multiple Myeloma." Blood 128, no. 22 (2016): 799. http://dx.doi.org/10.1182/blood.v128.22.799.799.

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Abstract Introduction According to the clonal evolution model, tumor progression proceeds in a branching rather than in a linear manner, leading to substantial clonal diversity and coexistence of genetically heterogeneous sets of subclones. Unlike many cancers, in which the evolutionary history can only be inferred from the established disease, Multiple Myeloma (MM) has well defined precursor states, which offer a unique opportunity to study the sequential evolution of the disease. In MM, multiple subclones can co-exist because they are of similar fitness, potentially interact with each other

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Cai, Shuyang, Honghu Li, Ruxiu Tie, Qian Luo, He Huang, and Jiahui Lu. "Clec16a-Mediated Mitophagy Modulates Zebrafish Definitive Hematopoiesis." Blood 144, Supplement 1 (2024): 30. https://doi.org/10.1182/blood-2024-198955.

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Hematopoietic stem and progenitor cells (HSPCs) generate all blood cell lineages throughout the lifespan of vertebrates. The emergence of HSPCs occurs through the hemogenic endothelial (HE) to hematopoietic transition (EHT) process, which is finely regulated by a variety of signaling pathways. Previous studies have highlighted the essential roles of pattern-recognition receptors such as Toll-like receptors, RIG-I-like receptors, and NOD-like receptors in EHT. However, whether members of the C-type Lectin Receptors (CLRs) family participate in vertebrate embryonic hematopoiesis remains unclear.

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Couch, Tyler A., Zachary C. Murphy, Michael Getman, Ryo Kurita, Yukio Nakamura, and Laurie A. Steiner. "Human Erythroblasts with c-Kit Activating Mutations Have Reduced Cell Culture Costs and Remain Capable of Terminal Maturation and Enucleation." Blood 132, Supplement 1 (2018): 2315. http://dx.doi.org/10.1182/blood-2018-99-117157.

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Abstract There is a constant need for red blood cells for transfusion therapy in the treatment of anemias and acute injury. As all blood products for transfusion come from donors, there are concerns over shortages and safety. Furthermore, many patients with transfusion-dependent anemias risk alloiumminization. The in vitro production of red blood cells would address these problems, especially as they can be genetically engineered to prevent alloimmunization. Numerous erythroid culture systems now exist for the in vitro production of red blood cells. Hematopoietic stem and progenitor cells (HSP

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El Hoss, Sara, Sylvie Cochet, Auria Godard, et al. "Fetal Hemoglobin Rescues Ineffective Erythropoiesis in Sickle Cell Disease." Blood 136, Supplement 1 (2020): 14–15. http://dx.doi.org/10.1182/blood-2020-137477.

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Sickle cell disease (SCD) is an autosomal hereditary recessive disorder caused by a point mutation in the β globin gene resulting in a Glu-to-Val substitution at the 6th position of the β globin protein. The resulting abnormal hemoglobin (HbS) polymerizes under hypoxic conditions driving red blood cell (RBC) sickling (Pauling et al., 1949). While pathobiology of circulating RBCs has been extensively analyzed in SCD, erythropoiesis is surprisingly poorly documented. In β-thalassemia, ineffective erythropoiesis is characterized by high levels of apoptotic erythroblasts during the late stages of

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Guruprasad, Puneeth, Alberto Carturan, Yunlin Zhang, et al. "Modulation of the Btla-HVEM Axis to Enhance CAR T Cell Immunotherapy Against Cancer." Blood 142, Supplement 1 (2023): 768. http://dx.doi.org/10.1182/blood-2023-189633.

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Introduction: The efficacy of adoptive T cell immunotherapies against cancer, such as chimeric antigen receptor (CAR) T cells, is severely blunted by the immunosuppressive tumor microenvironment (TME). We sought to investigate the role of the TME in cancer resistance to effector T cells in order to define actionable targets to enhance CAR T cell immunotherapies. We initially used Hodgkin lymphoma (HL) as an ideal tumor model since it is characterized by a TME that is profoundly infiltrated by immunosuppressive cells, and then we expanded our findings to multiple cancer models. We first sought

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Cai, Shuyang, Honghu Li, Ruxiu Tie, et al. "Nlrc3 Signaling Is Indispensable for Hematopoietic Stem Cell Emergence Via Notch Signaling in Vertebrates." Blood 142, Supplement 1 (2023): 2675. http://dx.doi.org/10.1182/blood-2023-186693.

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Hematopoietic stem and progenitor cells (HSPCs) generate all the lineages of blood cells throughout the lifespan of vertebrates. The emergence of HSPCs occurs through the hemogenic endothelial (HE) to hematopoietic transition (EHT) process, which is finely tuned by a variety of signaling pathways. Previous studies have emerged the essential roles of pattern-recognition receptors such as Toll-like receptors and RIG-I-like receptors in EHT. However, whether the nucleotide-binding domain leucine-rich repeat (NLR) containing family members participate in vertebrate embryonic hematopoiesis remains

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Heatley, Susan L., Elyse C. Page, Laura N. Eadie, et al. "Modeling Relapsed, Refractory Acute Lymphoblastic Leukemia from a Child with Neurofibromatosis." Blood 138, Supplement 1 (2021): 1317. http://dx.doi.org/10.1182/blood-2021-150086.

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Abstract Neurofibromatosis type 1 (NF-1) is an autosomal dominant disorder affecting approximately 1:3000 individuals globally. While approximately 50% are familial, with over 3000 causative germline variants in the neurofibromatosis (NF1) gene identified, the remainder occur sporadically. These mutations lead to haploinsufficiency of NF1 and neurofibromin, a tumor suppressor and important negative regulator of RAS signaling. Children with NF-1 have a higher risk of developing juvenile myelomonocytic leukemia and acute myeloid leukemia, but rarely develop acute lymphoblastic leukemia (ALL). A

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Ghobadi, Armin, Ibrahim Aldoss, Shannon L. Maude, et al. "Phase 1/2 Dose-Escalation/Dose-Expansion Study of Anti-CD7 Allogeneic CAR-T Cells (WU-CART-007) in Relapsed or Refractory (R/R) T-Cell Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (T-ALL/LBL)." Blood 142, Supplement 1 (2023): 770. http://dx.doi.org/10.1182/blood-2023-178723.

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T-ALL/LBL are challenging hematologic cancers with high rates of relapse and mortality in both children and adults. Despite success in B-cell malignancies, development of CAR-T cell therapy for T cell malignancies has been complicated by induction of fratricide and risk of malignant cell contamination of the drug product in the autologous setting. WU-CART-007 is a CD7-targeted CAR-T cell products with CRISPR/Cas9 deletion of CD7 and T-cell receptor alpha constant (TRAC), to prevent fratricide and enable the use of healthy donor allogeneic T-cells, respectively (Leedom et al. ASH 2021). This of

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