Ribosomal proteins and human diseases: molecular mechanisms and targeted therapy
Ribosome biogenesis and protein synthesis are fundamental rate-limiting steps for cell
growth and proliferation. The ribosomal proteins (RPs), comprising the structural parts of the …
growth and proliferation. The ribosomal proteins (RPs), comprising the structural parts of the …
Context is everything: aneuploidy in cancer
U Ben-David, A Amon - Nature Reviews Genetics, 2020 - nature.com
Cancer is driven by multiple types of genetic alterations, which range in size from point
mutations to whole-chromosome gains and losses, known as aneuploidy. Chromosome …
mutations to whole-chromosome gains and losses, known as aneuploidy. Chromosome …
Molecular international prognostic scoring system for myelodysplastic syndromes
E Bernard, H Tuechler, PL Greenberg… - NEJM …, 2022 - evidence.nejm.org
Background Risk stratification and therapeutic decision-making for myelodysplastic
syndromes (MDS) are based on the International Prognostic Scoring System–Revised (IPSS …
syndromes (MDS) are based on the International Prognostic Scoring System–Revised (IPSS …
Translation of cytoplasmic UBA1 contributes to VEXAS syndrome pathogenesis
MA Ferrada, S Savic, DO Cardona… - Blood, The Journal …, 2022 - ashpublications.org
Somatic mutations in UBA1 cause vacuoles, E1 ubiquitin-activating enzyme, X-linked,
autoinflammatory somatic (VEXAS) syndrome, an adult-onset inflammatory disease with an …
autoinflammatory somatic (VEXAS) syndrome, an adult-onset inflammatory disease with an …
Randomized comparison of low dose cytarabine with or without glasdegib in patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic …
JE Cortes, FH Heidel, A Hellmann, W Fiedler, BD Smith… - Leukemia, 2019 - nature.com
Glasdegib is a Hedgehog pathway inhibitor. This phase II, randomized, open-label,
multicenter study (ClinicalTrials. gov, NCT01546038) evaluated the efficacy of glasdegib …
multicenter study (ClinicalTrials. gov, NCT01546038) evaluated the efficacy of glasdegib …
[HTML][HTML] Luspatercept in patients with lower-risk myelodysplastic syndromes
P Fenaux, U Platzbecker, GJ Mufti… - … England Journal of …, 2020 - Mass Medical Soc
Background Patients with anemia and lower-risk myelodysplastic syndromes in whom
erythropoiesis-stimulating agent therapy is not effective generally become dependent on red …
erythropoiesis-stimulating agent therapy is not effective generally become dependent on red …
Myelodysplastic syndromes: 2023 update on diagnosis, risk‐stratification, and management
G Garcia‐Manero - American journal of hematology, 2023 - Wiley Online Library
Disease overview The myelodysplastic syndromes (MDS) are a very heterogeneous group
of myeloid disorders characterized by peripheral blood cytopenias and increased risk of …
of myeloid disorders characterized by peripheral blood cytopenias and increased risk of …
Phase I First-in-Human Dose Escalation Study of the oral SF3B1 modulator H3B-8800 in myeloid neoplasms
DP Steensma, M Wermke, VM Klimek, PL Greenberg… - Leukemia, 2021 - nature.com
We conducted a phase I clinical trial of H3B-8800, an oral small molecule that binds Splicing
Factor 3B1 (SF3B1), in patients with MDS, CMML, or AML. Among 84 enrolled patients (42 …
Factor 3B1 (SF3B1), in patients with MDS, CMML, or AML. Among 84 enrolled patients (42 …
A phase 3 randomized study of 5-azacitidine maintenance vs observation after transplant in high-risk AML and MDS patients
This study investigated the efficacy and safety of azacitidine maintenance in the
posttransplant setting based on the encouraging phase 1/2 reports for azacitidine …
posttransplant setting based on the encouraging phase 1/2 reports for azacitidine …
Myeloablative versus reduced-intensity hematopoietic cell transplantation for acute myeloid leukemia and myelodysplastic syndromes
BL Scott, MC Pasquini, BR Logan, J Wu… - Journal of Clinical …, 2017 - ascopubs.org
Purpose The optimal regimen intensity before allogeneic hematopoietic cell transplantation
(HCT) is unknown. We hypothesized that lower treatment-related mortality (TRM) with …
(HCT) is unknown. We hypothesized that lower treatment-related mortality (TRM) with …