Sickle cell disease (SCD) is one of the most common haemoglobinopathies worldwide, with up to 70 % of global SCD annual births occurring in sub-Saharan Africa. Reports have shown that 50 to 80 % of affected children in these countries die annually. Efforts geared towards understanding and controlling HbF production in SCD patients could lead to strategies for effective control of globin gene expression and therapeutic approaches that could be beneficial to individuals with haemoglobinopathies. Hemopoietic stem cells (HSCs) are characterized by a specific miRNA signature in every state of differentiation. The role of miRNAs has become evident both in the maintenance of the "stemness" and in the early induction of differentiation by modulation of the expression of the master pluripotency genes and during early organogenesis. miRNAs are extra regulatory mechanisms in hematopoietic stem cells (HSCs) via influencing transcription profiles together with transcript stability. miRNAs have been reported to be used to reprogram primary somatic cells toward pluripotency. Their involvement in cell editing holds the potential for therapy for many genetic diseases. This review provides a snapshot of miRNA involvement in cell fate decisions, haemoglobin induction pathway, and their journey as some emerge prime targets for therapy in beta haemoglobinopathies.
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http://dx.doi.org/10.2174/1574888X17666220221104711 | DOI Listing |
Biomedicines
January 2025
Clinical Research Center, Jiangnan University Medical Center, 68 Zhongshan Road, Wuxi 214002, China.
Acute myeloid leukemia (AML) is an aggressive cancer with variable treatment responses. While clinical factors such as age and genetic mutations contribute to prognosis, recent studies suggest that CT attenuation scores may also predict treatment outcomes. This study aims to develop a nomogram combining clinical and CT-based factors to predict treatment response and guide personalized therapy for AML patients.
View Article and Find Full Text PDFCardiovasc Diabetol
January 2025
Department of Hematology, Endocrinology and Metabolism, Niigata University Faculty of Medicine, 1-757 Asahimachi-dori, Chuo Ward, Niigata, 951-8510, Japan.
Background: Previous studies demonstrated that diabetes remission can occur during intensive intervention and in real-world settings. However, the impact of diabetes remission in real-world settings on the incidence of cardiovascular disease (CVD) remains unclear.
Methods: This retrospective cohort study included 299,967 individuals aged 20-72 years who underwent multiple checkups between 2008 and 2020 and completed ≥ 3 years of follow-up.
BMJ
January 2025
Ministry of Education Key Laboratory of Metabolism and Molecular Medicine, Department of Endocrinology and Metabolism, Zhongshan Hospital, Fudan University, Shanghai, China.
Objective: To assess the effect of dapagliflozin plus calorie restriction on remission of type 2 diabetes.
Design: Multicentre, double blind, randomised, placebo controlled trial.
Setting: 16 centres in mainland China from 12 June 2020 to 31 January 2023.
ACS Pharmacol Transl Sci
January 2025
Guangdong-Hong Kong-Macao Joint Laboratory for New Drug Screening, School of Pharmacy, The Chinese University of Hong Kong, Shatin 999077, Hong Kong SAR, P. R. China.
Erythroferrone (ERFE) has emerged as a potential biomarker for the erythropoiesis response following recombinant human erythropoietin (rHuEPO) treatment. While the association between ERFE and hemoglobin (HGB) response to rHuEPO is well-established in nonanemic conditions, such correlation and ERFE kinetics in anemic states remain unclear. We employed two rat models of anemia, chronic kidney disease (CKD) anemia and chemotherapy-induced anemia (CIA), to determine ERFE kinetics and its correlation with HGB responses after rHuEPO administration.
View Article and Find Full Text PDFJ Minim Invasive Gynecol
January 2025
Division of Urogynecology, Department of Obstetrics and Gynaecology, Mount Sinai Hospital, University of Toronto, Toronto, Ontario, Canada; Present Affiliation (not associated with study): Department of Obstetrics and Gynecology, Cambridge Memorial Hospital, Cambridge, Ontario, Canada.
Objective: To determine the efficacy of intravenous (IV) tranexamic acid (TXA) in reducing blood loss and blood transfusion among women undergoing total colpocleisis.
Design: Double-blind, randomized, placebo-controlled trial.
Setting: Tertiary academic urogynecology practice.
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