☆ 4.7 Review

Current status and future perspectives in targeted therapy of NPM1-mutated AML

LEUKEMIA (2022)

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Review Oncology

Menin Inhibitors in Acute Myeloid Leukemia-What Does the Future Hold?

Mahesh Swaminathan et al.

Summary: Menin inhibitors are a class of drugs targeting NPM1 mutant and KMT2Ar acute leukemias, with the aim of inducing differentiation, downregulating critical gene expression, and providing a survival advantage. Multiple clinical trials are ongoing, and preliminary results show promising effects and tolerable toxicity.

CANCER JOURNAL (2022)

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Article Oncology

Impact of FLT3 Mutation on Outcomes after Venetoclax and Azacitidine for Patients with Treatment-Naive Acute Myeloid Leukemia

Marina Konopleva et al.

Summary: The efficacy and safety of venetoclax + azacitidine in patients with FLT3-mutant acute myeloid leukemia were evaluated. The results showed significant differences in composite complete remission rates and overall survival between patients treated with venetoclax + azacitidine and those treated with azacitidine alone. These findings suggest that venetoclax + azacitidine may be of importance in the treatment of patients with FLT3 mutations.

CLINICAL CANCER RESEARCH (2022)

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Article Oncology

Effective Menin inhibitor-based combinations against AML with MLL rearrangement or NPM1 mutation (NPM1c)

Warren Fiskus et al.

Summary: Treatment with Menin inhibitor disrupts the interaction between Menin and MLL1 or MLL1-fusion protein, inhibiting AML cell survival and inducing differentiation. Combining Menin inhibitor with other drugs enhances the therapeutic efficacy against AML cells.

BLOOD CANCER JOURNAL (2022)

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Article Oncology

Pembrolizumab and decitabine for refractory or relapsed acute myeloid leukemia

Meghali Goswami et al.

Summary: This study aimed to investigate the feasibility and efficacy of the combination of PD-1 blockade and hypomethylating therapy in patients with R-AML. The results showed that this combination therapy achieved disease stability or better response in some patients. Moreover, clonal T cell expansions and immunological changes were observed during treatment, which may be associated with treatment-related adverse events.

JOURNAL FOR IMMUNOTHERAPY OF CANCER (2022)

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Article Biochemistry & Molecular Biology

EAPB0503, an Imidazoquinoxaline Derivative Modulates SENP3/ARF Mediated SUMOylation, and Induces NPM1c Degradation in NPM1 Mutant AML

Hala Skayneh et al.

Summary: Nucleophosmin-1 (NPM1) is a pleiotropic protein involved in various cellular processes. In acute myeloid leukemia (AML), NPM1 frequently mutates and exhibits aberrant cytoplasmic localization. Therefore, finding therapeutic approaches to modulate NPM1c post-translational modifications is of great significance in the management of AML patients.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2022)

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Article Oncology

Use of Venetoclax in Patients with Relapsed or Refractory Acute Myeloid Leukemia: The PETHEMA Registry Experience

Jorge Labrador et al.

Summary: The effectiveness of venetoclax (VEN) in relapsed or refractory acute myeloid leukemia (RR-AML) has not been well established. This study found that the combination of venetoclax with azacitidine had a higher complete remission rate compared to combination with decitabine and low-dose cytarabine. Mutated NPM1 was associated with increased complete remission rate.

CANCERS (2022)

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Article Hematology

Treatment-free remission after ceasing venetoclax-based therapy in patients with acute myeloid leukemia

Chong Chyn Chua et al.

Blood Advances (2022)

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Article Hematology

Enhanced stimulation of antigen-specific immune responses against nucleophosmin 1 mutated acute myeloid leukaemia by an anti-programmed death 1 antibody

Jochen Greiner et al.

Summary: This study investigated immune responses against NPM1 and other leukaemia-associated antigens (LAA) in AML patients and found that the anti-PD-1 antibody nivolumab enhances immune response against stem cell-like cells. It suggests that NPM1 mutated AML patients could benefit from treatment with anti-PD-1 and NPM1-specific immunotherapy.

BRITISH JOURNAL OF HAEMATOLOGY (2022)

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Review Oncology

The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms

Joseph D. Khoury et al.

Summary: This paper summarizes the new WHO classification scheme for myeloid and histiocytic/dendritic neoplasms, emphasizing the refinement of diagnostic criteria and the importance of actionable biomarkers, while maintaining global applicability.

LEUKEMIA (2022)

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Article Multidisciplinary Sciences

Memory-like NK cells armed with a neoepitope-specific CAR exhibit potent activity against NPM1 mutated acute myeloid leukemia

Han Dong et al.

Summary: Acute myeloid leukemia (AML) is difficult to treat due to a lack of tumor-specific targets for immune-based therapies. Recent studies have shown that natural killer (NK) cells exhibit innate memory and enhanced antitumor activity when activated with IL-12 and IL-18. This study demonstrates that arming CIML NK cells with a neoepitope-specific CAR significantly improves their antitumor responses to NPM1-mutated AML and avoids off-target toxicity.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2022)

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Article Hematology

Venetoclax induces rapid elimination of NPM1 mutant measurable residual disease in combination with low-intensity chemotherapy in acute myeloid leukaemia

Ing S. Tiong et al.

Summary: This study demonstrates the significant efficacy of venetoclax-based therapy in reducing the risk of relapse in patients with persistent or rising NPM1(mut) MRD.

BRITISH JOURNAL OF HAEMATOLOGY (2021)

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Article Oncology

Venetoclax Increases Intratumoral Effector T Cells and Antitumor Efficacy in Combination with Immune Checkpoint Blockade

Frederick J. Kohlhapp et al.

Summary: Venetoclax, a selective BCL2 inhibitor, can enhance the anticancer efficacy of immune checkpoint inhibitors by increasing PD-1+ T effector memory cells in mouse models. It does not impair human T-cell function and can provide a survival advantage in effector T cells, indicating its potential in combination with ICIs for cancer therapy.

CANCER DISCOVERY (2021)

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Article Engineering, Biomedical

CAR-T cells targeting a nucleophosmin neoepitope exhibit potent specific activity in mouse models of acute myeloid leukaemia

Guozhu Xie et al.

Summary: Therapies using CAR-T cells targeting tumor-specific driver gene mutations show potent and specific cytotoxic activity against acute myeloid leukemia, potentially avoiding harmful effects on normal tissues.

NATURE BIOMEDICAL ENGINEERING (2021)

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Meeting Abstract Hematology

CD123 and CD33 Co-Targeting By Balanced Signaling on CAR-CIK Cells Reduces Potential Off-Target Toxicity While Preserving the Anti-Leukemic Activity of Acute Myeloid Leukemia

Vincenzo Maria Perriello et al.

BLOOD (2021)

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Review Chemistry, Medicinal

A Box of Chemistry to Inhibit the MEN1 Tumor Suppressor Gene Promoting Leukemia

Ezgi Ozyerli-Goknar et al.

Summary: The study introduces a new class of PPI inhibitors targeting the interaction between Menin and MLL proteins to treat MLL/KMT2A-rearranged leukemia, with thienopyrimidine compounds showing promising results in late-phase clinical trials. These compounds exhibit good tolerability and minimal side effects, suggesting a potential novel targeted therapy for leukemia patients.

CHEMMEDCHEM (2021)

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Article Oncology

Dactinomycin induces complete remission associated with nucleolar stress response in relapsed/refractory NPM1-mutated AML

Ilaria Gionfriddo et al.

Summary: Relapsed or refractory AML patients with NPM1 mutations face challenges in treatment, particularly in elderly and unfit individuals. Dactinomycin, a low-cost chemotherapeutic agent, shows potential as an alternative treatment option, possibly through its association with nucleolar stress. Further investigation in larger clinical studies is warranted.

LEUKEMIA (2021)

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Article Multidisciplinary Sciences

The tumor therapy landscape of synthetic lethality

Biyu Zhang et al.

Summary: Synthetic lethality is becoming an important cancer therapeutic paradigm, but comprehensive selective treatment opportunities for various tumors have not been fully explored. The Synthetic Lethality Knowledge Graph (SLKG) integrates data on different tumors, drugs, and drug targets to provide therapy options for synthetic lethality and synthetic dosage lethality, prioritizing the identification of repurposable drug candidates and combinations with supporting evidence for novel tumor therapy discovery.

NATURE COMMUNICATIONS (2021)

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Article Oncology

CD123 Is Consistently Expressed on NPM1-Mutated AML Cells

Vincenzo Maria Perriello et al.

Summary: One-third of adult AML patients have NPM1 mutations, with high expression of CD123 identified as a potential target for therapy in NPM1-mutated leukemic cells, particularly in CD34(+)CD38(-) cells. Targeting CD123 may be effective for treating NPM1-mutated AML, especially in combination with FLT3 mutations.

CANCERS (2021)

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Article Hematology

Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML

Maximilian Stahl et al.

Summary: Combination therapy of venetoclax is effective in many patients with relapsed or refractory AML, with azacitidine+venetoclax showing higher response rates compared to low-dose cytarabine+venetoclax. Clinical and molecular characteristics of patients may predict treatment outcomes in this population.

BLOOD ADVANCES (2021)

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Article Hematology

Venetoclax enhances T cell-mediated antileukemic activity by increasing ROS production

Jong Bok Lee et al.

Summary: The combination therapy of Venetoclax and azacytidine directly activates T cells, increases reactive oxygen species generation, and enhances cytotoxicity against AML cells. Azacytidine also activates the STING/cGAS pathway, rendering AML cells more susceptible to T cell-mediated cytotoxicity.

BLOOD (2021)

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Article Oncology

An HLA-A*11:01-Binding Neoantigen from Mutated NPM1 as Target for TCR Gene Therapy in AML

Dyantha I. van der Lee et al.

Summary: AML is an aggressive hematological malignancy with a poor prognosis, requiring new and effective therapies; approximately 30-35% of AML cases have a frameshift mutation in the dNPM1 gene, potentially creating targets for immunotherapy.

CANCERS (2021)

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Article Oncology

Diagnostic and therapeutic pitfalls in NPM1-mutated AML: notes from the field

Brunangelo Falini et al.

Summary: Mutations of Nucleophosmin (NPM1) are common in adult AML and are recognized as a distinct entity. Evaluation of NPM1 and FLT3 status is important for risk stratification and monitoring of MRD. Combining MRD monitoring with the ELN prognostication model can help guide therapeutic decisions for NPM1-mutated AML.

LEUKEMIA (2021)

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Letter Hematology

Menin inhibition decreases Bcl-2 and synergizes with venetoclax in NPM1/FLT3-mutated AML

Bing Z. Carter et al.

BLOOD (2021)

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Article Oncology

A phase 1b/2 study of azacitidine with PD-L1 antibody avelumab in relapsed/refractory acute myeloid leukemia

Kapil Saxena et al.

Summary: Patients with relapsed/refractory acute myeloid leukemia (AML) have limited treatment options. The combination of azacitidine and the anti-PD-L1 immune checkpoint inhibitor avelumab showed limited clinical activity. High expression of PD-L2 on bone marrow blasts may contribute to resistance to anti-PD-L1 therapy in AML.

CANCER (2021)

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Article Oncology

Venetoclax Combined With FLAG-IDA Induction and Consolidation in Newly Diagnosed and Relapsed or Refractory Acute Myeloid Leukemia

Courtney D. DiNardo et al.

Summary: The combination of fludarabine, cytarabine, granulocyte colony-stimulating factor, and idarubicin with venetoclax showed to be an effective treatment regimen in both newly diagnosed and relapsed or refractory AML patients, resulting in deep remissions and high rates of successful transplantation.

JOURNAL OF CLINICAL ONCOLOGY (2021)

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Review Oncology

Therapeutic implications of menin inhibition in acute leukemias

Ghayas C. Issa et al.

Summary: Menin inhibitors are novel targeted agents currently being developed for the treatment of genetically defined subsets of acute leukemia, showing promising early results in specific leukemia types, such as NPM1 mutations. These inhibitors target various gene regulatory roles of menin in leukemogenesis and are being investigated in clinical studies for relapsed acute leukemias.

LEUKEMIA (2021)

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Article Oncology

A 2:1 randomized, open-label, phase II study of selinexor vs. physician's choice in older patients with relapsed or refractory acute myeloid leukemia

Kendra Sweet et al.

Summary: Selinexor, a selective inhibitor of nuclear export, showed promising activity in patients with acute myeloid leukemia (AML). However, in patients aged >= 60 years with relapsed/refractory (R/R) AML, there was no significant difference in overall survival between selinexor and physician's choice treatment. Despite some patients responding well to selinexor, there was a higher incidence of adverse events in the selinexor group.

LEUKEMIA & LYMPHOMA (2021)

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Article Oncology

Actinomycin D Targets NPM1c-Primed Mitochondria to Restore PML-Driven Senescence in AML Therapy

Hsin-Chieh Wu et al.

Summary: NPM1c mutation affects mitochondrial biogenesis and PML NBs, while ActD targets mitochondria to generate ROS, promoting PML NB biogenesis and restoring senescence. Dual targeting of mitochondria with ActD and venetoclax enhances their anti-AML activities.

CANCER DISCOVERY (2021)

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Article Oncology

Phase II Trial of Pembrolizumab after High-Dose Cytarabine in Relapsed/Refractory Acute Myeloid Leukemia

Joshua F. Zeidner et al.

Summary: The study demonstrated that pembrolizumab following high-dose cytarabine in relapsed/refractory AML patients was well-tolerated and feasible, showing promising clinical activity, especially in refractory AML and those receiving treatment as first salvage regimen. Further exploration of pembrolizumab and other immune-checkpoint blockade strategies post cytotoxic chemotherapy is recommended in AML.

BLOOD CANCER DISCOVERY (2021)

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Review Biochemistry & Molecular Biology

Small molecules in targeted cancer therapy: advances, challenges, and future perspectives

Lei Zhong et al.

Summary: Targeted therapeutic drugs have become mainstream cancer treatments due to their advantages in efficacy and safety, but still face challenges such as low response rate and drug resistance. A comprehensive review was conducted on small-molecule targeted anti-cancer drugs to promote their development, discussing current challenges and providing insights and perspectives for future research and development.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2021)

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Letter Biophysics

Favorable outcomes for allogeneic hematopoietic cell transplantation in elderly patients with NPM1-mutated and FLT3-ITD-negative acute myeloid leukemia

Ibrahim Aldoss et al.

BONE MARROW TRANSPLANTATION (2020)

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Article Oncology

Genetic characterization of ABT-199 sensitivity in human AML

Richard Bisaillon et al.

LEUKEMIA (2020)

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Article Oncology

Selinexor in combination with decitabine in patients with acute myeloid leukemia: results from a phase 1 study

Bhavana Bhatnagar et al.

LEUKEMIA & LYMPHOMA (2020)

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Review Pharmacology & Pharmacy

Epigenetic strategies synergize with PD-L1/PD-1 targeted cancer immunotherapies to enhance antitumor responses

Xi Chen et al.

ACTA PHARMACEUTICA SINICA B (2020)

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Article Oncology

Resistance Mechanisms to SYK Inhibition in Acute Myeloid Leukemia

Anjali Cremer et al.

CANCER DISCOVERY (2020)

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Letter Hematology

Venetoclax and arsenic showed synergistic anti-leukemia activity in vitro and in vivo for acute myeloid leukemia with the NPM1 mutation

Hong-Hu Zhu et al.

AMERICAN JOURNAL OF HEMATOLOGY (2020)

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Article Hematology

Cytogenetics and mutations could predict outcome in relapsed and refractory acute myeloid leukemia patients receiving BCL-2 inhibitor venetoclax

Yu-Wen Wang et al.

ANNALS OF HEMATOLOGY (2020)

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Article Hematology

Molecular patterns of response and treatment failure after frontline venetoclax combinations in older patients with AML

C. D. DiNardo et al.

BLOOD (2020)

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Article Hematology

Mutational profile and benefit of gemtuzumab ozogamicin in acute myeloid leukemia

Elise Fournier et al.

BLOOD (2020)

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Article Medicine, Research & Experimental

Menin inhibitor MI-3454 induces remission in MLL1-rearranged and NPM1-mutated models of leukemia

Szymon Klossowski et al.

JOURNAL OF CLINICAL INVESTIGATION (2020)

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Article Oncology

Gemtuzumab Ozogamicin in NPM1-Mutated Acute Myeloid Leukemia: Early Results From the Prospective Randomized AMLSG 09-09 Phase III Study

Richard F. Schlenk et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Review Oncology

Recent drug approvals for newly diagnosed acute myeloid leukemia: gifts or a Trojan horse?

Elihu Estey et al.

LEUKEMIA (2020)

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Article Multidisciplinary Sciences

Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemia

Hannah J. Uckelmann et al.

SCIENCE (2020)

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Article Hematology

Venetoclax plus LDAC for newly diagnosed AML ineligible for intensive chemotherapy: a phase 3 randomized placebo-controlled trial

Andrew H. Wei et al.

BLOOD (2020)

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Editorial Material Oncology

It's All About MEis: Menin-MLL Inhibition Eradicates NPM1-Mutated and MLL-Rearranged Acute Leukemias in Mice

Michael C. Gundry et al.

CANCER CELL (2020)

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Review Medicine, Research & Experimental

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Luca Vago et al.

JOURNAL OF CLINICAL INVESTIGATION (2020)

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Article Multidisciplinary Sciences

Composition-dependent thermodynamics of intracellular phase separation

Joshua A. Riback et al.

NATURE (2020)

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Article Hematology

Synergistic targeting of FLT3 mutations in AML via combined menin-MLL and FLT3 inhibition

Margarita M. Dzama et al.

BLOOD (2020)

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Review Hematology

NPM1-mutated acute myeloid leukemia: from bench to bedside

Brunangelo Falini et al.

BLOOD (2020)

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Letter Hematology

A Phase II study of selinexor plus cytarabine and idarubicin in patients with relapsed/refractory acute myeloid leukaemia

Walter Fiedler et al.

BRITISH JOURNAL OF HAEMATOLOGY (2020)

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Review Biochemistry & Molecular Biology

Nucleophosmin in Its Interaction with Ligands

Ilaria Cela et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2020)

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Article Oncology

Chemotherapy and Venetoclax in Elderly Acute Myeloid Leukemia Trial (CAVEAT): A Phase Ib Dose-Escalation Study of Venetoclax Combined With Modified Intensive Chemotherapy

Chong Chyn Chua et al.

JOURNAL OF CLINICAL ONCOLOGY (2020)

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Review Oncology

Proteolysis targeting chimeras (PROTACs) are emerging therapeutics for hematologic malignancies

Yonghan He et al.

JOURNAL OF HEMATOLOGY & ONCOLOGY (2020)

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Article Hematology

Impact of gemtuzumab ozogamicin on MRD and relapse risk in patients with NPM1-mutated AML: results from the AMLSG 09-09 trial

Silke Kapp-Schwoerer et al.

BLOOD (2020)

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Article Hematology

Combinatorial treatment with menin and FLT3 inhibitors induces complete remission in AML models with activating FLT3 mutations

Hongzhi Miao et al.

BLOOD (2020)

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Correction Oncology

Immunogenomic Landscape of Hematological Malignancies (vol 38, pg 380, 2020)

Olli Dufva et al.

CANCER CELL (2020)

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Article Medicine, General & Internal

Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

C. D. DiNardo et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

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Article Oncology

Entospletinib in Combination with Induction Chemotherapy in Previously Untreated Acute Myeloid Leukemia: Response and Predictive Significance of HOXA9 and MEIS1 Expression

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CLINICAL CANCER RESEARCH (2020)

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Article Cell Biology

Immune landscapes predict chemotherapy resistance and immunotherapy response in acute myeloid leukemia

Jayakumar Vadakekolathu et al.

SCIENCE TRANSLATIONAL MEDICINE (2020)

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Approval of tagraxofusp-erzs for blastic plasmacytoid dendritic cell neoplasm

Naveen Pemmaraju et al.

BLOOD ADVANCES (2020)

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Article Hematology

How I diagnose and treat NPM1-mutated AML

Brunangelo Falini et al.

BLOOD (2020)

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Article Hematology

Venetoclax response is enhanced by selective inhibitor of nuclear export compounds in hematologic malignancies

Melissa A. Fischer et al.

BLOOD ADVANCES (2020)

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Article Hematology

Outcomes of older patients with NPM1-mutated AML: current treatments and the promise of venetoclax-based regimens

Curtis A. Lachowiez et al.

BLOOD ADVANCES (2020)

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Article Oncology

Venetoclax Combined With Low-Dose Cytarabine for Previously Untreated Patients With Acute Myeloid Leukemia: Results From a Phase Ib/II Study

Andrew H. Wei et al.

JOURNAL OF CLINICAL ONCOLOGY (2019)

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Article

Characterization and dynamics of specific T cells against nucleophosmin-1 (NPM1)-mutated peptides in patients with NPM1-mutated acute myeloid leukemia

Fabio Forghieri et al.

Oncotarget (2019)

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Article Biochemistry & Molecular Biology

High PD-L1 Expression Predicts for Worse Outcome of Leukemia Patients with Concomitant NPM1 and FLT3 Mutations

Barbora Brodska et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

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Article Multidisciplinary Sciences

Acute myeloid leukemia immunopeptidome reveals HLA presentation of mutated nucleophosmin

Rupa Narayan et al.

PLOS ONE (2019)

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Review Oncology

Recent drug approvals for acute myeloid leukemia

Catherine Lai et al.

JOURNAL OF HEMATOLOGY & ONCOLOGY (2019)

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Article Medicine, Research & Experimental

The tumor inflammation signature (TIS) is associated with anti-PD-1 treatment benefit in the CERTIM pan-cancer cohort

Diane Damotte et al.

JOURNAL OF TRANSLATIONAL MEDICINE (2019)

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Chromatin-bound CRM1 recruits SET-Nup214 and NPM1c onto HOX clusters causing aberrant HOX expression in leukemia cells

Masahiro Oka et al.

ELIFE (2019)

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Article Oncology

A Menin-MLL Inhibitor Induces Specific Chromatin Changes and Eradicates Disease in Models of MLL-Rearranged Leukemia

Andrei V. Krivtsov et al.

CANCER CELL (2019)

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Article Medicine, Research & Experimental

Mutated nucleophosmin 1 as immunotherapy target in acute myeloid leukemia

Dyantha van der Lee et al.

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Article Oncology

Efficacy, Safety, and Biomarkers of Response to Azacitidine and Nivolumab in Relapsed/Refractory Acute Myeloid Leukemia: A Nonrandomized, Open-Label, Phase II Study

Naval Daver et al.

CANCER DISCOVERY (2019)

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Article Hematology

Real-world experience of venetoclax with azacitidine for untreated patients with acute myeloid leukemia

Amanda C. Winters et al.

BLOOD ADVANCES (2019)

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Review Biochemistry & Molecular Biology

PROTACs: great opportunities for academia and industry

Xiuyun Sun et al.

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Self-interaction of NPM1 modulates multiple mechanisms of liquid-liquid phase separation

Diana M. Mitrea et al.

NATURE COMMUNICATIONS (2018)

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Mutant NPM1 Maintains the Leukemic State through HOX Expression

Lorenzo Brunetti et al.

CANCER CELL (2018)

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Inhibition of Amino Acid Metabolism Selectively Targets Human Leukemia Stem Cells

Courtney L. Jones et al.

CANCER CELL (2018)

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Venetoclax with azacitidine disrupts energy metabolism and targets leukemia stem cells in patients with acute myeloid leukemia

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Association of HLA class I type with prevalence and outcome of patients with acute myeloid leukemia and mutated nucleophosmin

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HOX gene expression predicts response to BCL-2 inhibition in acute myeloid leukemia

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KPT-8602, a second-generation inhibitor of XPO1-mediated nuclear export, is well tolerated and highly active against AML blasts and leukemia-initiating cells

J. Etchin et al.

LEUKEMIA (2017)

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Letter Hematology

Acute myeloid leukemia with mutated nucleophosmin 1: an immunogenic acute myeloid leukemia subtype and potential candidate for immune checkpoint inhibition

Jochen Greiner et al.

HAEMATOLOGICA (2017)

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Review Pharmacology & Pharmacy

Small-Molecule Screens: A Gateway to Cancer Therapeutic Agents with Case Studies of Food and Drug Administration-Approved Drugs

Nathan P. Coussens et al.

PHARMACOLOGICAL REVIEWS (2017)

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Letter Hematology

Acute myeloid leukemia with mutated nucleophosmin 1: an immunogenic acute myeloid leukemia subtype and potential candidate for immune checkpoint inhibition

Jochen Greiner et al.

HAEMATOLOGICA (2017)

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Article Oncology

Deguelin induced differentiation of mutated NPM1 acute myeloid leukemia in vivo and in vitro

Xia Zhang et al.

ANTI-CANCER DRUGS (2017)

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Article Oncology

Persistent Immune Stimulation Exacerbates Genetically Driven Myeloproliferative Disorders via Stromal Remodeling

Claudio Tripodo et al.

CANCER RESEARCH (2017)

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Article Hematology

A phase 1 clinical trial of single-agent selinexor in acute myeloid leukemia

Ramiro Garzon et al.

BLOOD (2017)

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Article Oncology

Imidazoquinoxaline derivative EAPB0503: A promising drug targeting mutant nucleophosmin 1 in acute myeloid leukemia

Ali I. Nabbouh et al.

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Article Oncology

Hoxa9 and Meis1 Cooperatively Induce Addiction to Syk Signaling by Suppressing miR-146a in Acute Myeloid Leukemia

Sebastian Mohr et al.

CANCER CELL (2017)

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Article Biochemistry & Molecular Biology

Coexisting Liquid Phases Underlie Nucleolar Subcompartments

Marina Feric et al.

CELL (2016)

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Article Oncology

Next-generation XPO1 inhibitor shows improved efficacy and in vivo tolerability in hematological malignancies

Z. A. Hing et al.

LEUKEMIA (2016)

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Assessment of Minimal Residual Disease in Standard-Risk AML

A. Ivey et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

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Article Medicine, General & Internal

Genomic Classification and Prognosis in Acute Myeloid Leukemia

Elli Papaemmanuil et al.

NEW ENGLAND JOURNAL OF MEDICINE (2016)

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Article Cell Biology

Anti-proliferative activity of the NPM1 interacting natural product avrainvillamide in acute myeloid leukemia

Vibeke Andresen et al.

CELL DEATH & DISEASE (2016)

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Article Multidisciplinary Sciences

A redox mechanism underlying nucleolar stress sensing by nucleophosmin

Kai Yang et al.

NATURE COMMUNICATIONS (2016)

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Article Oncology

Targeting Chromatin Regulators Inhibits Leukemogenic Gene Expression in NPM1 Mutant Leukemia

Michael W. M. Kuhn et al.

CANCER DISCOVERY (2016)

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Review Oncology

Molecules that target nucleophosmin for cancer treatment: an update

Adele Di Matteo et al.

ONCOTARGET (2016)

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Nucleophosmin integrates within the nucleolus via multi-modal interactions with proteins displaying R-rich linear motifs and rRNA

Diana M. Mitrea et al.

ELIFE (2016)

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Review Biochemistry & Molecular Biology

Inhibiting cancer cell hallmark features through nuclear export inhibition

Qingxiang Sun et al.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2016)

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Article Biochemistry & Molecular Biology

Interactions of the Natural Product (+)-Avrainvillamide with Nucleophosmin and Exportin-1 Mediate the Cellular Localization of Nucleophosmin and its AML-Associated Mutants

Herschel Mukherjee et al.

ACS CHEMICAL BIOLOGY (2015)

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Article Hematology

Arsenic trioxide and all-trans retinoic acid target NPM1 mutant oncoprotein levels and induce apoptosis in NPM1-mutated AML cells

Maria Paola Martelli et al.

BLOOD (2015)

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Article Hematology

Retinoic acid and arsenic trioxide trigger degradation of mutated NPM1, resulting in apoptosis of AML cells

Hiba El Hajj et al.

BLOOD (2015)

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Article Oncology

Epigenomic analysis of the HOX gene loci reveals mechanisms that may control canonical expression patterns in AML and normal hematopoietic cells

D. H. Spencer et al.

LEUKEMIA (2015)

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Letter Medicine, General & Internal

Dactinomycin in NPM1-Mutated Acute Myeloid Leukemia

Brunangelo Falini et al.

NEW ENGLAND JOURNAL OF MEDICINE (2015)

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Article Multidisciplinary Sciences

Altered HLA Class I Profile Associated with Type A/D Nucleophosmin Mutation Points to Possible Anti-Nucleophosmin Immune Response in Acute Myeloid Leukemia

Katerina Kuzelova et al.

PLOS ONE (2015)

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Article Hematology

Deguelin, a selective silencer of the NPM1 mutant, potentiates apoptosis and induces differentiation in AML cells carrying the NPM1 mutation

Sha Yi et al.

ANNALS OF HEMATOLOGY (2015)

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Review Oncology

Mouse models of NPM1-mutated acute myeloid leukemia: biological and clinical implications

P. Sportoletti et al.

LEUKEMIA (2015)

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Review Hematology

The past and future of CD33 as therapeutic target in acute myeloid leukemia

George S. Laszlo et al.

BLOOD REVIEWS (2014)

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Article Oncology

VISTA Is an Immune Checkpoint Molecule for Human T Cells

J. Louise Lines et al.

CANCER RESEARCH (2014)

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Article Oncology

Addition of gemtuzumab ozogamicin to induction chemotherapy in adult patients with acute myeloid leukaemia: a meta-analysis of individual patient data from randomised controlled trials

Robert K. Hills et al.

LANCET ONCOLOGY (2014)

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Article Multidisciplinary Sciences

Structural polymorphism in the N-terminal oligomerization domain of NPM1

Diana M. Mitrea et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

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Letter Hematology

Immune responses against the mutated region of cytoplasmatic NPM1 might contribute to the favorable clinical outcome of AML patients with NPM1 mutations (NPM1mut)

Jochen Greiner et al.

BLOOD (2013)

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Article Oncology

Donor Lymphocyte Infusion Induces Polyspecific CD8+ T-Cell Responses With Concurrent Molecular Remission in Acute Myeloid Leukemia With NPM1 Mutation

Susanne Hofmann et al.

JOURNAL OF CLINICAL ONCOLOGY (2013)

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Article Medicine, General & Internal

Genomic and Epigenomic Landscapes of Adult De Novo Acute Myeloid Leukemia

Timothy J. Ley et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

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Review Biochemistry & Molecular Biology

Nucleophosmin mutations in acute myeloid leukemia: A tale of protein unfolding and mislocalization

Luca Federici et al.

PROTEIN SCIENCE (2013)

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Article Biochemistry & Molecular Biology

Menin: a scaffold protein that controls gene expression and cell signaling

Smita Matkar et al.

TRENDS IN BIOCHEMICAL SCIENCES (2013)

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Article Hematology

Mutated regions of nucleophosmin 1 elicit both CD4+ and CD8+ T-cell responses in patients with acute myeloid leukemia

Jochen Greiner et al.

BLOOD (2012)

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Review Oncology

Discovery of small molecule cancer drugs: Successes, challenges and opportunities

Swen Hoelder et al.

MOLECULAR ONCOLOGY (2012)

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Article Biochemistry & Molecular Biology

Menin-MLL inhibitors reverse oncogenic activity of MLL fusion proteins in leukemia

Jolanta Grembecka et al.

NATURE CHEMICAL BIOLOGY (2012)

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Article Hematology

Targeting levels or oligomerization of nucleophosmin 1 induces differentiation and loss of survival of human AML cells with mutant NPM1

Ramesh Balusu et al.

BLOOD (2011)

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Review Hematology

Acute myeloid leukemia with mutated nucleophosmin (NPM1): Any hope for a targeted therapy?

Brunangelo Falini et al.

BLOOD REVIEWS (2011)

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Article Hematology

High CD33 expression levels in acute myeloid leukemia cells carrying the nucleophosmin (NPM1) mutation

Maria Stefania De Propris et al.

HAEMATOLOGICA-THE HEMATOLOGY JOURNAL (2011)

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Article Hematology

CD34+ cells from AML with mutated NPM1 harbor cytoplasmic mutated nucleophosmin and generate leukemia in immunocompromised mice

Maria Paola Martelli et al.

BLOOD (2010)

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Article Biochemistry & Molecular Biology

Chemotherapeutic Drugs Inhibit Ribosome Biogenesis at Various Levels

Kaspar Burger et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2010)

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Editorial Material Biotechnology & Applied Microbiology

Targeted cancer therapies

Saurabh Aggarwal

NATURE REVIEWS DRUG DISCOVERY (2010)

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Article Biochemistry & Molecular Biology

Nucleolar targeting: the hub of the matter

Edward Emmott et al.

EMBO REPORTS (2009)

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Review Oncology

Altered nucleophosmin transport in acute myeloid leukaemia with mutated NPM1: molecular basis and clinical implications

B. Falini et al.

LEUKEMIA (2009)

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Review Oncology

Targeting cancer with small molecule kinase inhibitors

Jianming Zhang et al.

NATURE REVIEWS CANCER (2009)

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Article Biochemistry & Molecular Biology

Structural Consequences of Nucleophosmin Mutations in Acute Myeloid Leukemia

Charles G. Grummitt et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2008)

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Article Biochemistry & Molecular Biology

NSC348884, a nucleophosmin inhibitor disrupts oligomer formation and induces apoptosis in human cancer cells

W. Qi et al.

ONCOGENE (2008)

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Article Multidisciplinary Sciences

Distinctive microRNA signature of acute myeloid leukemia bearing cytoplasmic mutated nucleophosmin

Ramiro Garzon et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

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Letter Oncology

In human genome, generation of a nuclear export signal through duplication appears unique to nucleophosmin (NPM1) mutations and is restricted to AML

A. Liso et al.

LEUKEMIA (2008)

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Letter Oncology

Nucleophosmin leukaemic mutants contain C-terminus peptides that bind HLA class I molecules

A. Liso et al.

LEUKEMIA (2008)

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Article Chemistry, Multidisciplinary

The natural product avrainvillamide binds to the oncoprotein nucleophosmin

Jeremy E. Wulff et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2007)

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Review Biochemistry & Molecular Biology

Hox genes in hematopoiesis and leukemogenesis

B. Argiropoulos et al.

ONCOGENE (2007)

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Review Oncology

Nucleophosmin and cancer

Silvia Grisendi et al.

NATURE REVIEWS CANCER (2006)

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Article Hematology

Both carboxy-terminus NES motif and mutated tryptophan(s) are crucial for aberrant nuclear export of nucleophosmin leukemic mutants in NPMc+ AML

Brunangelo Falini et al.

BLOOD (2006)

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Article Hematology

Acute myeloid leukemia bearing cytoplasmic nucleophosmin (NPMc+ AML) shows a distinct gene expression profile characterized by up-regulation of genes involved in stem-cell maintenance

M Alcalay et al.

BLOOD (2005)

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Article Medicine, General & Internal

Cytoplasmic nucleophosmin in acute myelogenous leukemia with a normal karyotype.

B Falini et al.

NEW ENGLAND JOURNAL OF MEDICINE (2005)

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Article Biochemistry & Molecular Biology

Menin associates with a trithorax family histone methyltransferase complex and with the Hoxc8 locus

CM Hughes et al.

MOLECULAR CELL (2004)

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