Optimized Chemical Modifications Enhance Lipid Nanoparticle-Mediated siRNA Silencing of YAP1 and WWTR1 [0.03%]
优化的化学修饰可增强脂质纳米颗粒介导的YAP1和WWTR1的siRNA沉默作用
Kaito Ueda,Tatsuki Sato,Jumpei Sasaki et al.
Kaito Ueda et al.
Lipid nanoparticles (LNPs) are clinically validated carriers for the delivery of small interfering RNA (siRNA). Their efficient tissue accessibility and cellular uptake complement conjugated siRNA approaches. While chemical modifications ar...
Bahareh Hosseinpour,Joshua O&#x;Grady,Neda Mohaghegh et al.
Bahareh Hosseinpour et al.
Aptamers are single-stranded synthetic oligonucleotides that bind noncovalently to targets with high affinity and selectivity. They are generated through an in vitro selection process known as Systematic Evolution of Ligands by Exponential ...
Exploring Clearance Pathways for GalNAc-siRNAs: Insights into Intracellular Kinetics and Therapeutic Implications [0.03%]
用于GalNAC-siRNA的清除途径研究：细胞内动力学及治疗意义的新见解
Madeline C Tompach,Kevin Craig,Shrutokirti De
Madeline C Tompach
Small interfering RNA (siRNA) represents a transformative therapeutic class that enables precise gene silencing through RNA interference (RNAi). N-acetyl galactosamine (GalNAc)-conjugated siRNAs have achieved remarkable clinical success wit...
Unraveling the Stereochemical Complexity of Phosphorothioate-Modified Oligonucleotides Using Analytical Technologies [0.03%]
利用分析技术解开硫代磷酸修饰寡核苷酸立体化学复杂性
Akanksha Manghrani,Maharshi C Patel,Frank Delaglio et al.
Akanksha Manghrani et al.
Oligonucleotide therapeutics are emerging as a promising modality for targeting disease-associated RNAs. Phosphorothioate (PS)-containing oligonucleotides have gained prominence due to their enhanced stability and pharmacodynamic properties...
Rat and Rabbit Whole-Embryo Culture as a New Approach Method for Unlabeled Therapeutic Antisense Oligonucleotide Hazard Identification with No Requirement for Microinjection or Assisted Transfection [0.03%]
新型无标记治疗性反义寡核苷酸风险识别方法——无需显微注射或辅助转染的整胚培养体系的建立与应用：以鼠和兔为例
Sara M Bender,Sharon Chapman,Sreenivas Nannapaneni et al.
Sara M Bender et al.
The developmental hazard screening of oligonucleotide therapeutics (ONTs) presents challenges due to their frequent lack of pharmacology in nonclinical species and embryo-fetal exposure is presumed to be limited in vivo. This study demonstr...
Programmable RNA Editing via Adenosine Deaminase Acting on RNA Enzymes: Current Advances and Clinical Potential [0.03%]
通过作用于RNA的腺苷脱氨酶实现的可编程RNA编辑：当前进展和临床潜力
Jinsil Kim,Hyuk Gyoon Lee,Minwook Shin
Jinsil Kim
Adenosine deaminase acting on RNA (ADAR)-mediated RNA editing has emerged as a powerful and precise technology for modifying RNA transcripts, enabling correction of disease-causing mutations without permanent changes to the genome. Recent a...
Efficient Downregulation of Flt-1 Mediated by Splice Switching ASO in Murine Endothelial Cells [0.03%]
高效下调小鼠内皮细胞中Flt-1的剪接开关ASO机制
Mathilde Blitek,Olivier Le Coz,Vincent Ogor et al.
Mathilde Blitek et al.
Impaired angiogenesis is a common feature of several pathological conditions, including neuromuscular disorders. Such vascular defects not only contribute to disease progression but also may compromise the efficacy of systemically delivered...
The Approval of Redemplo for Familial Chylomicronemia Syndrome and the Many Flavors of GalNAc-Oligonucleotides [0.03%]
阿利兰莫尔的获批及多种类型的半乳糖凝集素-寡核苷酸结构式
Julia F Alterman,Katherine Y Gross,Anastasia Khvorova
Julia F Alterman
In the fourth quarter of 2025, a press release announced the approval of the eighth small interfering RNA (siRNA)-based therapeutic. Redemplo (plozasiran), developed by Arrowhead Pharmaceuticals, is the third oligonucleotide-based medicine ...
Reduced Genotoxicity Testing Is Possible for Noncoding Oligonucleotide-Based Therapeutics Containing Well-Characterized Modifications: A European Regulatory Perspective [0.03%]
欧洲监管机构视角下的非编码寡核苷酸类药物遗传毒性试验减免路径指导建议
Clara Stock,Britt Duijndam,Christine L E Siezen et al.
Clara Stock et al.
There is a current lack of harmonized regulatory guidance in evaluating the genotoxic potential of oligonucleotide-based therapeutics (ONTs). In particular, guidance has not established the circumstances under which it is acceptable to devi...
Conjugated Antisense Oligonucleotides for Skipping of Duchenne Muscular Dystrophy Exon 53: A Cautionary Study [0.03%]
跳过杜氏肌营养不良外显子53的共轭反义寡核苷酸：一项警惕性研究
Emma T Groenwold,Alicia Montulet,Tiberiu Stan et al.
Emma T Groenwold et al.
Exon skipping antisense oligonucleotides (AONs) have been extensively studied as a promising method of treating Duchenne muscular dystrophy (DMD), yet the clinical efficacy of the conditionally approved AONs still remains low. Using phospho...