Applying Synthetic Nucleic Acid Therapeutics to Gene Activation for Haploinsufficient Diseases [0.03%]
合成核酸治疗技术激活基因治疗剂量限制性单倍型不足疾病
Victor Tse,David R Corey
Victor Tse
Haploinsufficient autosomal dominant diseases are due to heterozygous mutations that cause inadequate protein expression. Compounds that increase expression of the wild-type allele would be one strategy for treating patients. Synthetic anti...
An RNA Aptamer Targeting PAI-1 That Restores tPA Activity, Unexpectedly Suppresses Cancer Cell Progression [0.03%]
一种意外地抑制癌细胞进展的结合PAI-1并恢复tPA活性的RNA适配体
Christopher Hu,Austin Schneidler,Mamadou Barry et al.
Christopher Hu et al.
Plasminogen activator inhibitor-1 (PAI-1), the primary physiological inhibitor of tissue-type plasminogen activator (tPA), is a key regulator of fibrinolysis. Elevated levels of PAI-1 are linked to thrombotic disorders and correlate with po...
Antisense Oligonucleotide Quantification via Splint-Ligation PCR Assay in Nonhuman Primate Central Nervous System Tissues and Biofluids [0.03%]
用于非人灵长类动物中枢神经系统组织和体液中反义寡核苷酸定量的接头连接PCR测定法
Thomas Jepp,Sarah Christian,Scott V Dindot
Thomas Jepp
Antisense oligonucleotides (ASOs) are chemically modified single-stranded oligonucleotides used to modulate the expression or processing of a target RNA transcript. The development of ASOs to treat human disease requires extensive preclinic...
Unveiling Liver Micro-Distribution: NanoSIMS Imaging Reveals Critical Intracellular Distribution of Chemically Modified Antisense Oligonucleotides [0.03%]
揭开肝脏微分布之谜：NanoSIMS成像揭示化学修饰反义寡核苷酸的细胞内关键分布规律
Hidenori Yasuhara,Kenta Kadotsuji,Kenichi Watanabe et al.
Hidenori Yasuhara et al.
Antisense oligonucleotides (ASOs) represent a promising class of therapeutic agents; yet, their efficacy and/or toxicity profiles are heavily dependent on their tissue distribution and cellular uptake. This study employs nanoscale secondary...
Class-Specific Adverse Events of Patients Treated with Small Interfering RNA Therapeutics: A Disproportionality Analysis of the United States Food and Drug Administration Adverse Event Reporting System Database Based on the MY FAERS Platform [0.03%]
基于MY FAERS平台的美国食品药品管理局不良事件报告系统数据库的小干扰rna药物类属不良反应信号分析
Ze Li,Xiaozhen Wang,Dandan Li et al.
Ze Li et al.
Small interfering RNA (siRNA) therapeutics represent a transformative class of drugs, but their class-specific adverse events (CAE-siRNA) remain incompletely characterized. This study aimed to identify and quantify CAE-siRNA associated with...
Targeting a Pathogenic Variant Creating an Upstream AUG in the ENG 5' Untranslated Region with Antisense Oligonucleotides Fails to Restore Protein Expression [0.03%]
反义寡核苷酸未能通过在ENG的5'非翻译区创建上游AUG来靶向致病突变以恢复蛋白质表达
Mathilde Doisy,Aris Gaci,Omar Soukarieh et al.
Mathilde Doisy et al.
Pathogenic variants creating upstream open reading frames (uORFs) in the 5' untranslated region (5'UTR) of the ENG gene can disrupt translation from the main ORF and contribute to hereditary hemorrhagic telangiectasia (HHT). This is the cas...
Immunogenicity Risk Assessment for Nucleic Acid Therapeutics: A Comprehensive Evaluation for ASO, siRNA, and Nonvaccine mRNA/LNP Therapies by the IQ Consortium [0.03%]
IQ联盟针对核酸药物的免疫原性风险评估：全面评估ASO、siRNA和非疫苗mRNA/LNP疗法的免疫原性风险
Joanna Grudzinska-Goebel,Manuela Braun,Lin-Zhi Chen et al.
Joanna Grudzinska-Goebel et al.
The emergence of nucleic acid (NA) therapeutics, including antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), which are usually delivered directly, and messenger RNAs (mRNAs), which are typically encapsulated in lipid nanop...
A Workflow for Transcriptome-Wide Assessment of Antisense Oligonucleotide Selectivity [0.03%]
反义寡核苷酸选择性评估的转录组级工作流程
Sagar S Damle,Andy Watt,Steven Kuntz et al.
Sagar S Damle et al.
Antisense oligonucleotides (ASOs) designed to recruit RNase H1 (gapmer ASOs) have been used successfully to downregulate the expression of therapeutic targets. Gapmer ASOs can be identified that selectively reduce the expression of transcri...
Melanie Sauer,Xavier Segarra-Visent,Leon Breuer et al.
Melanie Sauer et al.
Cryopreservation is a routine step in the manufacturing process of adoptive cell therapies (ACT), providing critical logistic flexibility. RNA interference (RNAi)-based therapies are increasingly being explored as enhancers or modulators of...