Spleen-targeted mRNA delivery via long-chain PEGylated lipids at low molar ratio enhances antitumor immunity against melanoma [0.03%]
通过低摩尔比的长链聚乙二醇化脂质将mRNA递送至脾脏，增强针对黑色素瘤的抗肿瘤免疫反应
Shiyu Liu,Longlong Zhang,Wenbo Wu et al.
Shiyu Liu et al.
Lipid nanoparticles (LNPs) are pivotal for mRNA delivery, yet predominant hepatic accumulation limits their therapeutic application in immunologically active sites like the spleen. Engineering LNPs for splenic mRNA delivery is therefore ess...
The 2025 Oligo Meeting in Budapest: Highlights of the 21st annual meeting of the oligonucleotide therapeutics society [0.03%]
第21届寡核苷酸治疗学会年会(Oligo 2025)会议纪要
Bruno M D C Godinho,Shuling Guo
Bruno M D C Godinho
Ribofuranose-based GalNAc-conjugated siRNA enhances the liver-targeted delivery and elicits robust RNAi-mediated gene silencing [0.03%]
以核糖呋喃糖为基础的GalNAC-siRNA能够增强肝靶向递送并有效激活RNA干扰介导的基因沉默作用
Ze-Ao Huang,Zhen-Min Li,Jie Wang et al.
Ze-Ao Huang et al.
The N-acetylgalactosamine (GalNAc)-conjugate delivery platform has emerged as a pivotal enabling technology for the clinical translation of oligonucleotide therapeutics. The development of GalNAc-conjugated small interfering RNA (siRNA) the...
Beyond the stop: Oxadiazole TRIDs restore LRBA protein expression in nonsense-driven primary immunodeficiency [0.03%]
停止密码子超越策略OXADIAZOLE-TRIDs治疗无义突变导致的LRBA缺陷原发性免疫缺陷病
Ignazio Fiduccia,Emanuele Vitale,Riccardo Varrica et al.
Ignazio Fiduccia et al.
Nonsense mutations are among the genetic causes of LRBA (lipopolysaccharide-responsive beige-like anchor) deficiency, a rare autosomal-recessive immunodeficiency disorder. These mutations introduce premature stop codons, leading to the loss...
Aptamer-based approaches for sensitive detection and epitope mapping of SARS-CoV-2 spike protein [0.03%]
用于检测和绘制SARS-CoV-2刺突蛋白表位的基于适配体的方法
Suttinee Poolsup,Elnaz Yaghoobi,Aliaksandra Radchanka et al.
Suttinee Poolsup et al.
The SARS-CoV-2 spike (S) protein, crucial for viral entry, remains a key target for diagnostics and therapeutics amid evolving variants. Here, we describe the selection and characterization of novel DNA aptamers targeting the S1 subunit, in...
IRES-based RNAs expressing co-stimulatory molecules: Promising candidates for cancer immunotherapy [0.03%]
基于IRES的表达共刺激分子的RNA：癌症免疫治疗的候选药物
Yun Ji Kim,Ji Young Bang,Hye-Won Yu et al.
Yun Ji Kim et al.
Optimizing co-stimulatory signaling to enhance T cell responses is central to effective antitumor immunity. In this study, we developed single-stranded RNAs (ssRNAs) utilizing the internal ribosome entry site (IRES) of encephalomyocarditis ...
Combining anti-gene γPNA with small molecules and RNA inhibitors: A strategy to enhance anti-tumor efficacy [0.03%]
反基因γPNA与小分子和RNA抑制剂结合：增强抗肿瘤疗效的策略
Sai Pallavi Pradeep,Brooke Elizabeth DiVasto,Peter M Glazer et al.
Sai Pallavi Pradeep et al.
Targeting genomic DNA to silence oncogenes is a promising strategy for cancer therapy. Gamma peptide nucleic acid (γPNA) targeting c-Myc genomic DNA has shown established efficacy in vitro and in multiple preclinical models. In this study,...
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides [0.03%]
通过皮下递送Gapmer反义寡核苷酸减少DUX4并改善肌肉功能
Aiping Zhang,Kenji Rowel Q Lim,Ze Chen et al.
Aiping Zhang et al.
Facioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of double homeodomain protein 4 (DUX4). The disease has no effective treatment. Previously we demonstrated effective DUX4 knockdown in vitro and in vivo using 2'...
AAV2-mediated intravitreal delivery of exon-specific U1 snRNA rescues optic neuropathy in familial dysautonomia [0.03%]
AAV2介导的针对外显子的U1 snRNA玻璃体内递送可缓解家族性自主神经失调病引起的视神经病变
Anil Chekuri,Krishnakanth Kondabolu,Emily G Kirchner et al.
Anil Chekuri et al.
Familial dysautonomia (FD) is a rare autosomal recessive neurodegenerative disorder caused by a splicing mutation in the ELP1 gene. It predominantly affects the sensory and autonomic nervous systems, with progressive vision loss due to opti...
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease [0.03%]
利用RNA靶向的CRISPR-Cas13和工程U1系统在Stargardt疾病中靶向ABCA4剪接变异体
Roxanne Hsiang-Chi Liou,Daniel Urrutia-Cabrera,Chia-Fei Liu et al.
Roxanne Hsiang-Chi Liou et al.
Dysregulation of the alternative splicing process results in aberrant mRNA transcripts, leading to dysfunctional proteins or nonsense-mediated decay that cause a wide range of mis-splicing diseases. Development of therapeutic strategies to ...