Results 41 to 50 of about 181,456 (327)

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

Unravelling the Complexity of the +33 C>G [HBB:c.-18C>G] Variant in Beta Thalassemia

Biomedicines
The +33 C>G variant [NM_000518.5(HBB):c.-18C>G] in the 5′ untranslated region (UTR) of the β-globin gene is described in the literature as both mild and silent, while it causes a phenotype of thalassemia intermedia in the presence of a severe β ...
Coralea Stephanou, Miranda Petrou, Petros Kountouris, Christiana Makariou, Soteroula Christou, Michael Hadjigavriel, Marina Kleanthous, Thessalia Papasavva   +7 more
doaj   +1 more source

Chronic hyperplastic anemia as an independent risk factor for atherosclerotic lesions: a lesson from thalassemia intermedia [PDF]

, 2010
Introduction. Cardiovascular involvement represents a well-known complication and the primary cause of mortality, both in transfusion-dependent beta thalassemia major (β-TM) and in transfusion-independent beta thalassemia intermedia (β-TI ...
Alessandra Spiga, Antonella Mandas, Fabrizio Angius, Maria Eliana Lai, Maria Paola Carta, Pier Luigi Cocco, Sandra Dessi, Stefania Vacquer   +7 more
core   +1 more source

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells. [PDF]

, 2015
Lentiviral vectors designed for the treatment of the hemoglobinopathies require the inclusion of regulatory and strong enhancer elements to achieve sufficient expression of the β-globin transgene.
Baldwin, Kismet M, Campo-Fernandez, Beatriz, Cooper, Aaron R, Hoban, Megan D, Hollis, Roger P, Kaufman, Michael L, Kohn, Donald B, Lumaquin, Dianne, Romero, Zulema, Senadheera, Shantha, Urbinati, Fabrizia, Wang, Xiaoyan, Wherley, Jennifer   +12 more
core   +4 more sources

Single‐Position Peptide Clustering for Peptidomics Reveals Novel Disease Biomarkers and Dysregulated Proteolytic Characteristics

Advanced Science, EarlyView.
A novel amino acid (aa)‐score‐based single‐position peptide clustering strategy is developed for peptidomics, enabling precise profiling of protein proteolysis in plasma from β‐thalassemia cohort. The method identifies new aa position‐based peptide cluster biomarkers validated by heavy‐labeled peptides, visualizes aggregated changes, uncovers disease ...
Na Li, Yaxin Zhu, Yumeng Yan, Jifeng Wang, Lili Niu, Xiang Ding, Mengmeng Zhang, Zhensheng Xie, Tanxi Cai, Xiaojing Guo, Jianming Luo, Peng An, Xiangqian Guo, Fuquan Yang   +13 more
wiley   +1 more source

Comparative efficacy of deferiprone, deferoxamine and combination of deferiprone and deferoxamine on serum ferritin value in Beta-Thalassemia patients [PDF]

, 2015
Background: Iron overload is a predictable and life-threatening complication in patients with thalassemia. Effective and convenient iron chelation remains one of the main targets of clinical management of thalassemia major.
Amlashi, H.M., Behnampour, N., Jahazi, A., Mirbehbahani, N.   +3 more
core   +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

Time to Do Something for Vitamin D Deficiency; A Review

Journal of Pediatrics Review, 2015
Context: Vitamin D deficiency is a common nutritional disorder in Iran. Vitamin D is an essential health factor from birth onward. This study was conducted to summarize epidemiologic researches regarding vitamin D deficiency in different parts of the ...
Mehrnoush Kosaryan, Mandana Zafari , Aili Aliasgharian, Masomeh Mosawi   +3 more
doaj   +3 more sources

INHERITED BONE MARROW FAILURE SYNDROMES WITH PANCYTOPAENIA [PDF]

European Medical Journal Hematology, 2014
Bone marrow failure (BMF) is characterised by a reduction in the effective production of mature erythrocytes, granulocytes, and platelets by the bone marrow that leads to peripheral blood pancytopaenia.
Sophia Delicou, Marianna Bellia, Theoni Kanellopoulou, Chryssanthi Kontaxi, Apostolos Zografakis, Konstantinos Maragkos   +5 more
doaj  

Targeted Carrier Screening for Thalassemia, Hereditary Deafness, and Spinal Muscular Atrophy: A Feasible Approach for Preventing Birth Defects in China's Community Healthcare System. [PDF]

Mol Genet Genomic Med
Current carrier screening primarily focuses on high detection rates and broad testing ranges. We approach the issue from the perspective of a community physician, evaluating the suitability of carrier screening based on factors such as cost, ease of report interpretation, and compliance issues.
Wang Z, Bai W, Cai X, Huang S, Zeng J, Liang X, Hong X.   +6 more
europepmc   +2 more sources