Results 51 to 60 of about 50,706 (199)

Nonsense-mediated decay mechanism is a possible modifying factor of clinical outcome in nonsense cd39 beta thalassemia genotype [PDF]

, 2012
Nonsense-mediated mRNA decay (NMD) is a surveillance system to prevent the synthesis of non-functional proteins. In β-thalassemia, NMD may have a role in clinical outcome.
ATTANASIO, Massimo, Calvaruso, G, Cassarà, F, Fecarotta, E, Fiorentino, G, Giambona, A, Maggio, A., Piazza, A, Renda, D, Renda, MC, Rigano, P, VITRANO, Angela   +11 more
core   +1 more source

Mutational analysis of the Janus kinase II (V617F) gene in patients with β-Thalassemia major

Zanco Journal of Pure and Applied Sciences, 2020
β-Thalassemia is a group of congenital hemolytic anemia that characterized by the underproduction of the indispensable hemoglobin molecule, the oxygen and carbon dioxide carrying protein inside the red cells.
Salar Adnan Ahmed
doaj   +3 more sources

Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study

Haematologica, 2019
β-thalassemia, a hereditary blood disorder caused by defective synthesis of hemoglobin β globin chains, leads to ineffective erythropoiesis and chronic anemia that may require blood transfusions.
Maria Domenica Cappellini, John Porter, Raffaella Origa, Gian Luca Forni, Ersi Voskaridou, Frédéric Galactéros, Ali T. Taher, Jean-Benoît Arlet, Jean-Antoine Ribeil, Maciej Garbowski, Giovanna Graziadei, Chantal Brouzes, Michaela Semeraro, Abderrahmane Laadem, Dimana Miteva, Jun Zou, Victoria Sung, Tatiana Zinger, Kenneth M. Attie, Olivier Hermine   +19 more
doaj   +1 more source

Inherited hemolytic disorders with high occurrence of b-thalassemia in Sindhi community of Jabalpur town in Madhya Pradesh, India [PDF]

, 2010
Hereditary hemolytic disorders such as hemoglobin disorders, β-thalassemia syndrome, G6PD deficiency, and ABO and Rhesus blood groups are the most common public health problems in India.
Balgir, RS
core   +1 more source

The Effect of Silymarin on Serum Concentration of Soluble Apoptosis Markers in β-Thalassemia Major Patients Receiving Desferrioxamine [PDF]

, 2015
Background: Despite appropriate chelation therapy with desferrioxamine, iron deposition in visceral organs causes tissue damage in thalassemia major patients.
Esmaeil, N., Gharagozloo, M., Ghatreh Samani, M., Mirmoghtadaei, M., Moayedi, B.   +4 more
core   +1 more source

Improved Outcomes for Older Children, Adolescents, and Young Adults With Neuroblastoma in the Post‐Immunotherapy Era: An Updated Report From the International Neuroblastoma Risk Group

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background We describe clinical and biologic characteristics of neuroblastoma in older children, adolescents, and young adults (OCAYA); describe survival outcomes in the post‐immunotherapy era; and identify if there is an age cut‐off that best discriminates outcomes.
Rebecca J. Deyell, Kavitha Srivatsa, Paola Angelini, Pei‐Chi Kao, Brady Payne, Arlene Naranjo, Sveva Castelli, Miho Kato, Miki Ohira, Ulriche Pöetschger, Yaël Mossé, Lucas Moreno, Meredith S. Irwin, Ramya Ramanujachar, Wendy B. London   +14 more
wiley   +1 more source

Long-term safety and erythroid response with luspatercept treatment in patients with β-thalassemia

Therapeutic Advances in Hematology, 2022
Background: β-thalassemia is a hereditary blood disorder resulting in ineffective erythropoiesis and anemia. Management of anemia with regular blood transfusions is associated with complications including iron overload.
Antonio Piga, Filomena Longo, Maria Rita Gamberini, Ersi Voskaridou, Paolo Ricchi, Vincenzo Caruso, Antonello Pietrangelo, Xiaosha Zhang, Jeevan K. Shetty, Kenneth M. Attie, Immacolata Tartaglione   +10 more
doaj   +1 more source

Potent Liver‐Tropic mRNA Lipid Nanoparticles: ApoE‐Mediated Delivery Through a Low‐Density Lipoprotein Receptor Independent Uptake Mechanism

Advanced Materials, EarlyView.
Helper and ionizable lipids play a crucial role in determining ApoE binding and subsequent liver tropism and LDLR‐mediated uptake. Ionizable lipids primarily govern the LDLR‐independent uptake pathway. This complementary interplay between lipid components ultimately governs LNP delivery performance and therapeutic efficacy in the liver.
Ashish Sarode, Christian Ortiz, Tadeh Derstepanian, Natalia Vargas‐Montoya, Priyal Patel, Nikita Khadse, Saikat Manna, Ryan Landis, Joseph Skaleski, Lianne Boeglin, Hongfeng Deng, Anusha Dias, Hong Wang, Debora Barreiros Petropolis, Barak Yahalom, Shrirang Karve, Frank DeRosa   +16 more
wiley   +1 more source

Discovery of a Novel DNMT1 Inhibitor with Improved Efficacy in Treating β‐Thalassemia

Advanced Science, EarlyView.
Context of Research: β‐thalassemia affects millions worldwide. DNMT inhibitors are effective HbF‐inducers that benefit patients with β‐thalassemia. Existing DNMT inhibitors are not approved for β‐thalassemia treatment due to dose‐limiting toxicity.What We Find: DMT207 traps DNMT1 into helix‐kinked inactive conformation and enhances its interaction with
Yijie Shen, Jiale Wei, Shibing Tang, Dongliang Wu, Liangyi Zong, Shuyuan Ma, Qing Xiong, Ruijie Gong, Siyuan Xu, Chuxuan Peng, Qin Feng, Songchen Liu, Qitong Liu, Yuhua Ye, Quan Zhao, Cheng Luo, Peng Huang, Zhihai Li, Xiangqian Kong, Xianjiang Lan   +19 more
wiley   +1 more source

Loss of SOCS1 in Donor T Cells Exacerbates Intestinal GVHD by Driving a Chemokine‐Dependent Pro‐Inflammatory Immune Microenvironment

Advanced Science, EarlyView.
T cell‐specific Socs1 knockout leads to inflammatory differentiation of CD8+ T cells, prompting the STAT1/2 complex to drive the activation of Ccl5, Ccr5, and Cxcr3, and promoting the skewing of monocytes toward a pro‐inflammatory M1 macrophage lineage.
Zhigui Wu, Bixia Wang, Xinya Jiang, Fangqing Zhang, Qianqian Huang, Xinyi Wu, Shuang Fan, Qi Zhang, Jingrui Zhou, Jianing Tang, Xiao‐Dong Mo, Yu Wang, Ying‐Jun Chang, Huidong Guo, Xiao‐Jun Huang   +14 more
wiley   +1 more source