Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications [0.03%]
通过战略化学修饰解缠和控制反义寡核苷酸的迟发型神经毒性
Takayuki Kuroda,Kotaro Yoshioka,Su Su Lei Mon et al.
Takayuki Kuroda et al.
Antisense oligonucleotides (ASOs) represent an attractive therapeutic approach for CNS disorders. However, ASO-induced neurotoxicity, especially late-onset adverse events, remains a crucial issue, leading to failures in clinical application...
MicroRNA chemical modifications in post-transcriptional gene silencing and human diseases [0.03%]
微小核糖核酸的化学修饰在转录后基因沉默及人类疾病中的作用
Amin Alaei,Pavan Kumar Kakumani
Amin Alaei
MicroRNAs (miRNAs) are small, non-coding RNAs that influence various cellular activities through post-transcriptional gene silencing. Recent research has shown that miRNA modifications, including N6-methyladenosine (m6A), 5-methylcytidine (...
Lipid nanoparticle-encapsulated microRNA-192: An anti-inflammatory adjuvant that enhances vaccine efficacy in aged mice [0.03%]
脂质纳米颗粒包封的微小核糖核酸-192：一种抗炎佐剂，可增强老年小鼠疫苗效力
Yuriko Takagi,Tasuku Nishimura,Suraiya Aktar et al.
Yuriko Takagi et al.
Proinflammatory cytokines are essential for initiating immune responses; however, excessive or aging-related chronic inflammation impairs immunity and reduces vaccine efficacy. In this study, we developed lipid nanoparticles (LNPs) encapsul...
Myomerger-derived peptide enhances skeletal muscle tropism and reduces liver transduction of lipid nanoparticles for gene delivery [0.03%]
一种新型肌肉趋向性肽可增强骨骼肌递送效率并降低脂质纳米颗粒的肝向分布
Jacqueline Ji,Eva Lipkow,Nicolas Anton et al.
Jacqueline Ji et al.
Lipid nanoparticles (LNPs) are emerging as nonviral vectors for gene therapy; yet, their strong liver tropism and lack of tissue specificity remain limiting. Here, we developed, through rational design, a skeletal muscle-targeted delivery p...
HLA-DQB1-mediated B cell-epithelial crosstalk drives EBV-associated inflammation in Hunner-type interstitial cystitis [0.03%]
HLA-DQB1介导的B细胞-上皮细胞串扰驱动Hunner型间质性膀胱炎相关EB病毒感染炎症
Shaik Ismail Mohammed Thangameeran,Jia-Fong Jhang,Yuan-Hong Jiang et al.
Shaik Ismail Mohammed Thangameeran et al.
Hunner-type interstitial cystitis (HIC) is a chronic condition marked by persistent pain and inflammation. To elucidate its immunogenetic drivers, we integrated bulk RNA sequencing and single-cell RNA sequencing datasets with targeted major...
The role of exosomes as oligonucleotide delivery system for managing α-synuclein in Parkinson's disease: A systematic review of in vivo studies [0.03%]
外泌体作为递送寡核苷酸管理帕金森病中α-突触核蛋白的系统：一项体内研究的系统评价
Soodeh Salarpour,Fatemeh Dourandish,Arsalan Salajegheh et al.
Soodeh Salarpour et al.
Although Parkinson's disease (PD) is primarily idiopathic, genetic mutations-accounting for approximately 5%-15% of cases with regional variability-have prompted the development of gene expression modulators, such as oligonucleotides, to ta...
mRNA-LNP vaccination orchestrates systemic immunity to control human papillomavirus-positive head and neck squamous cell carcinoma [0.03%]
基于 mRNA 的疫苗通过系统性免疫来控制人类乳头瘤病毒阳性头颈鳞癌
Ke Qiu,Minzi Mao,Yao Song et al.
Ke Qiu et al.
Messenger RNA (mRNA) vaccines have demonstrated significant potential in cancer immunotherapy by activating both innate and adaptive immunity. However, the detailed cellular and molecular dynamics underpinning these systemic immune response...
The dysregulation score method identifies epigenetic regulator genes that predict cancer prognosis and efficiency of cancer immunotherapy [0.03%]
一种失调评分方法可识别出可预测癌症预后和癌症免疫治疗效率的表观遗传调控基因
Jie Lyu,Hao Zhang,Jinjin Zhong et al.
Jie Lyu et al.
Epigenetic mechanisms play a crucial role in gene expression regulation during the initiation and progression of cancer. Despite this, over 600 epigenetic regulator (ER) genes, which are responsible for the reading, writing, and erasing of ...
Rethinking CRISPR delivery for liver-targeted gene editing: The case for spatially fractionated intra-arterial approaches [0.03%]
重新思考用于肝靶向基因编辑的CRISPR输送方法：空间分段动脉内给药方法的优势案例分析
Abdallah Salemdawod,Piotr Walczak,Miroslaw Janowski
Abdallah Salemdawod
Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice [0.03%]
AAV9-U7介导的mdx52小鼠第51外显子跳跃使大脑部分恢复Dp427，但未能改善行为反应
Ophélie Vacca,Amel Saoudi,Mathilde Doisy et al.
Ophélie Vacca et al.
The mdx52 mouse model exhibits a common mutation profile associated with brain involvement in Duchenne muscular dystrophy (DMD), characterized by heightened anxiety, fearfulness, and impaired associative fear learning. Deletion of exon 52 d...