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Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Haemophilia
January 2025
Medicine and Pathology, Georgetown University, Washington, District of Columbia, USA.
Introduction: Gene editing therapies offer the possibility of substantial improvement in treatment and quality of life for people with haemophilia (PWH) in a landscape of dynamic therapeutic advancement. Developing a common and understandable language to discuss gene editing will be essential to ensure these treatments can be deployed in a safe and effective manner with fully informed and shared decision-making between healthcare professionals (HCPs) and PWH. A lexicon explaining and clarifying key concepts is one potential tool to address these aims.
View Article and Find Full Text PDFThe Multifaceted Roles of MicroRNA-181 in Stem Cell Differentiation and Cancer Stem Cell Plasticity.
Cells
January 2025
CHU Sainte-Justine Research Center, Université de Montréal, Montreal, QC H3T 1C5, Canada.
Stem cells are undifferentiated or partially differentiated cells with an extraordinary ability to self-renew and differentiate into various cell types during growth and development. The epithelial-mesenchymal transition (EMT), a critical developmental process, enhances stem cell-like properties in cells, and is associated with both normal stem cell function and the formation of cancer stem cells. Cell stemness and the EMT often coexist and are interconnected in various contexts.
View Article and Find Full Text PDFPericardial effusion cytology: malignancy rates, patterns of metastasis, comparison with pericardial window, and genomic correlates.
J Am Soc Cytopathol
December 2024
Department of Pathology, Beth Israel Deaconess Medical Center and Harvard Medical School, Boston, Massachusetts. Electronic address:
Introduction: Cytologic evaluation of pericardial fluid is essential for diagnosing malignant pericardial effusions secondary to metastatic disease and for guiding appropriate clinical management; however, large cohort and up-to-date studies on malignancy rates and distribution of primary tumor sites is lacking.
Materials And Methods: A retrospective analysis of pericardial fluid specimens from 2 large academic medical centers over a 10-year period was conducted. Clinical and specimen characteristics were correlated with cytologic diagnoses, and compared with surgical pathology pericardial specimens when available.
An Unexpected Case of Clear Cell Adenocarcinoma of the Cervix in a Non-Diethylstilbestrol-Exposed Postmenopausal Woman.
Cureus
December 2024
Department of Family Medicine, Corewell Health Beaumont Grosse Pointe Hospital, Roseville, USA.
Clear cell adenocarcinoma of the cervix (CCAC) is a rare subtype of cervical adenocarcinoma. It has been linked to intrauterine exposure to diethylstilbestrol (DES) but can happen in non-DES-exposed patients, albeit less commonly. Presentation is largely vaginal bleeding, emphasizing the importance of considering CCAC in the differential of abnormal vaginal bleeding despite the tumor's rarity.
View Article and Find Full Text PDFA phase II study of ramucirumab and somatostatin analog therapy in patients with advanced neuroendocrine tumors.
Oncologist
January 2025
Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA 02215, United States.
Objectives: Well-differentiated neuroendocrine tumors (NET) are highly vascular tumors characterized by their expression of vascular endothelial growth factor (VEGF). This trial investigated the activity of ramucirumab, a monoclonal antibody that targets VEGF receptor-2 (VEGFR-2) and inhibits activity of VEGF, in combination with somatostatin analog therapy in patients (pts) with advanced extra-pancreatic NET.
Methods: We conducted a single-arm phase II trial enrolling pts with advanced, progressive extra-pancreatic NET.
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