Sequence-Controlled Spherical Nucleic Acids: Gene Silencing, Encapsulation, and Cellular Uptake [0.03%]
序列受控的球形核酸：基因沉默、包封作用和细胞摄取
Sepideh Kaviani,Hassan H Fakih,Jathavan Asohan et al.
Sepideh Kaviani et al.
Antisense oligonucleotides (ASOs) can predictably alter RNA processing and control protein expression; however, challenges in the delivery of these therapeutics to specific tissues, poor cellular uptake, and endosomal escape have impeded pr...
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics [0.03%]
核酸治疗剂临床前开发中使用的实验模型系统
Haiyan Zhou,Virginia Arechavala-Gomeza,Alejandro Garanto
Haiyan Zhou
Preclinical evaluation of nucleic acid therapeutics (NATs) in relevant experimental model systems is essential for NAT drug development. As part of COST Action "DARTER" (Delivery of Antisense RNA ThERapeutics), a network of researchers in t...
Positive Allosteric Modulation of Antithrombin's Inhibitory Activity by RNA Aptamers [0.03%]
RNA适配体对抗凝血酶活性的正向别构调节作用
Khalequz Zaman,Adi Breitman,Isa Malik et al.
Khalequz Zaman et al.
The leading cause of death in adults in the United States is cardiovascular disease, with mortality and morbidity mainly attributed to thromboembolism. Heparin is the most common therapy used for treating venous and arterial thrombosis. Hep...
Leon M Larcher,Ianthe L Pitout,Niall P Keegan et al.
Leon M Larcher et al.
Nucleic acids drugs have been proven in the clinic as a powerful modality to treat inherited and acquired diseases. However, key challenges including drug stability, renal clearance, cellular uptake, and movement across biological barriers ...
Complement C3d/C4d Deposition on Platelets Correlates with 2'-O-Methoxyethyl Antisense Oligonucleotide-Induced Thrombocytopenia in Monkeys [0.03%]
补体C3d/C4d沉积在血小板上与猴子中反义寡核苷酸诱导的血小板减少症相关性研究
Lijiang Shen,Andrea Wong,Satoru Oneda et al.
Lijiang Shen et al.
2'-O-Methoxyethyl antisense oligonucleotide (2'-MOE ASO)-induced severe thrombocytopenia (TCP) [platelet (PLT) count 5 mg/kg/week). The potential mechanisms for TCP were studied using the Mauritian-sourced cynomolgus monkeys, which were sho...
Next Generation Exon 51 Skipping Antisense Oligonucleotides for Duchenne Muscular Dystrophy [0.03%]
杜氏肌营养不良外显子51跳读反义寡核苷酸的下一代疗法研究
Judith van Deutekom,Chantal Beekman,Suzanne Bijl et al.
Judith van Deutekom et al.
In the last two decades, antisense oligonucleotides (AONs) that induce corrective exon skipping have matured as promising therapies aimed at tackling the dystrophin deficiency that underlies the severe and progressive muscle fiber degenerat...
Rapid and Reliable Quantification of Prime Editing Targeting Within the Porcine ABCA4 Gene Using a BRET-Based Sensor [0.03%]
基于BRET的传感器在猪ABCA4基因质粒转染效率及编辑位点定量中的应用研究
Tobias Wimmer,Hannah Sawinski,Anne M Urban et al.
Tobias Wimmer et al.
Stargardt disease (STGD) leads to blindness in children and young adults. So far, no curative therapy is available and gene augmentation therapies have not yet advanced to the clinics, in part, due to the limited packaging capacity of adeno...
Aerosolized Pulmonary Delivery of mRNA Constructs Attenuates Severity of Escherichia coli Pneumonia in the Rat [0.03%]
雾化吸入mRNA缓释技术可减轻大肠杆菌肺炎感染病情严重程度
Sean D McCarthy,Christopher B Rohde,Matt Angel et al.
Sean D McCarthy et al.
Acute respiratory distress syndrome (ARDS), a rapid onset inflammatory lung disease with no effective specific therapy, typically has pathogenic etiology termed pneumonia. In previous studies nuclear factor-κB (NF-κB) inhibitor α super-r...
Considerations for the Terminal Sterilization of Oligonucleotide Drug Products [0.03%]
寡核苷酸药物产品的终端灭菌考虑因素
Daniel Paul DeCollibus,Justin Searcy,Anna Tivesten et al.
Daniel Paul DeCollibus et al.
A primary function of the parenteral drug product manufacturing process is to ensure sterility of the final product. The two most common methods for sterilizing parenteral drug products are terminal sterilization (TS), whereby the drug prod...
Systematic Analysis of Chemical Modifications of Phosphorothioate Antisense Oligonucleotides that Modulate Their Innate Immune Response [0.03%]
系统分析环糊精化学修饰的磷硫酰亚胺反义寡核苷酸对其固有免疫反应的调节作用
Adam J Pollak,Luyi Zhao,Stanley T Crooke
Adam J Pollak
While rare, some gapmer phosphorothioate (PS) antisense oligonucleotides (ASOs) can induce a noncanonical TLR9-dependent innate immune response. In this study, we performed systematic analyses of the roles of PS ASO backbone chemistry, 2' m...