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http://dx.doi.org/10.1016/j.kint.2025.07.013 | DOI Listing |
Kidney Int
September 2025
Department of Nephrology and Medical Intensive Care, Charité-Universitätsmedizin Berlin, Berlin, Germany. Electronic address:
Zhonghua Xue Ye Xue Za Zhi
July 2025
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing 100730, China.
To evaluate the efficacy and safety of luspatercept combined with roxadustat in patients with refractory low-risk myelodysplastic neoplasms with ring sideroblasts (MDS-RS) patients. In this single-center, prospective, randomized controlled trial, patients with refractory MDS-RS were randomly assigned in a 1:2 ratio to receive either combination therapy (luspatercept + roxadustat) or luspatercept monotherapy. The primary endpoint was erythroid response at 12 weeks, while secondary endpoints included erythroid response at 24 weeks, achievement of transfusion independence ≥8 weeks within the first 12 weeks, and other hematologic indicators.
View Article and Find Full Text PDFScience
August 2025
Raina Biosciences, Cambridge, MA, USA.
Despite the success of mRNA COVID-19 vaccines, extending this modality to more diseases necessitates substantial enhancements. We present GEMORNA, a generative RNA model that utilizes Transformer architectures tailored for mRNA coding sequences (CDSs) and untranslated regions (UTRs), to design novel mRNAs with enhanced expression and stability. GEMORNA-designed full-length mRNAs exhibited up to a 41-fold increase in firefly luciferase expression compared to an optimized benchmark in vitro.
View Article and Find Full Text PDFCancer Discov
August 2025
Department of Pediatric Oncology/Hematology, Charité-Universitätsmedizin Berlin, corporate member of Freie Universität Berlin and Humboldt Universität zu Berlin, Berlin, Germany.
Unlabelled: Extrachromosomal DNA (ecDNA) amplification enhances intercellular oncogene dosage variability and accelerates tumor evolution by violating foundational principles of genetic inheritance through its asymmetric mitotic segregation. Spotlighting high-risk neuroblastoma, we demonstrate how ecDNA amplification undermines the clinical efficacy of current therapies in cancers with extrachromosomal MYCN amplification. Integrating theoretical models of oncogene copy number-dependent fitness with single-cell ecDNA quantification and phenotype analyses, we reveal that ecDNA copy-number heterogeneity drives phenotypic diversity and determines treatment sensitivity through mechanisms unattainable by chromosomal oncogene amplification.
View Article and Find Full Text PDFJ Med Case Rep
July 2025
Student Research Committee, School of Medicine, Iran University of Medical Sciences, Tehran, Iran.
Background: Myomatous erythrocytosis syndrome is an uncommon paraneoplastic syndrome of erythrocytosis associated with benign uterine leiomyomas. Fewer than 70 cases have been reported globally, and clinical awareness remains limited.
Case Presentation: We report the case of a 45-year-old Middle Eastern premenopausal woman who presented with a several-month history of abdominal distension and a hemoglobin level of 22.