An unbiased whole-genome open reading frame overexpression screen identifies B3GALT2, a novel inducer of cellular ASO activity [0.03%]
无偏倚的基因组开放阅读框过表达筛选鉴定出B3GALT2，这是一种新的细胞ASO活性诱导剂
Julia Hesselmann,Carolina Hager,Liza Malong et al.
Julia Hesselmann et al.
Antisense oligonucleotides (ASOs) are a promising class of therapeutics made of chemically modified synthetic single-stranded nucleic acid molecules, yet their clinical translation is often hindered by challenges in cellular uptake and deli...
Agnieszka Łoboda,Jeffrey S Chamberlain,Józef Dulak
Agnieszka Łoboda
Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder caused by mutations in the DMD gene, which encodes dystrophin, an essential structural muscle protein. Currently, there are no cures for DMD, and available therapies p...
A single-chain mRNA vaccine co-expressing GPC and NP provides complete protection against lethal Dabie bandavirus challenge in mice [0.03%]
一种表达GPC和NP的单链mRNA疫苗可完全保护小鼠免受致命的大别山蝙蝠病毒攻击
Entao Li,Haoyi He,Xiaoping Guo et al.
Entao Li et al.
Severe fever with thrombocytopenia syndrome (SFTS) is an emerging viral hemorrhagic fever caused by Dabie bandavirus (DBV), with a fatality rate of up to 30%. However, no approved vaccines or specific therapeutics against the virus are avai...
Suxiang Chen,Bal Hari Poudel,Rakesh Naduvile Veedu
Suxiang Chen
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells [0.03%]
反义寡核苷酸靶向E3连接酶RFFL可增强CFTR调节剂在囊性纤维化原发支气管上皮细胞中的疗效
Daichi Hinata,Yukari Kai,Ryosuke Fukuda et al.
Daichi Hinata et al.
Cystic fibrosis (CF) is most commonly caused by the ΔF508 mutation in the CFTR gene, leading to misfolding and degradation of the CFTR protein. Although CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) provide clinical benefi...
SCAD: A modular platform for efficient delivery of duplex RNA to the CNS and beyond [0.03%]
用于高效递送双链RNA到中枢神经系统及其更广泛区域的模块化平台
Moorim Kang,Wei-Hsiang Lin,Yichen Li et al.
Moorim Kang et al.
Oligonucleotide therapeutics-including antisense oligonucleotides and duplex RNAs such as small interfering RNAs, small activating RNAs, and microRNAs-hold immense potential for treating both genetic and acquired diseases by modulating gene...
A human-specific RPGR isoform and a clinically approved Rho/ROCK inhibitor ameliorate defects associated with RPGR dysfunction [0.03%]
一种人类特有的RPGR剪接变异体及一种临床获批的Rho/ ROCK抑制剂可改善RPGR功能障碍相关缺陷
Muhammad Usman,Paul Atigbire,Dennis Kastrati et al.
Muhammad Usman et al.
Pathogenic variants in the RPGR gene are the primary cause of photoreceptor degeneration in X-linked retinitis pigmentosa (RP). Previous studies have linked RPGR dysfunction to defects in ciliary structure and actin turnover. RPGR encodes t...
Systemic miR-26a deficiency attenuates pulmonary fibrosis via PTEN upregulation and downstream TIMP-1 suppression [0.03%]
miR-26a缺乏通过上调PTEN和下游TIMP-1抑制作用缓解肺纤维化
Arisa Hamada,Kiyofumi Shimoji,Taku Nakashima et al.
Arisa Hamada et al.
Several microRNAs (miRNAs) have been implicated in the pathophysiology of pulmonary fibrosis; however, the detailed mechanisms remain unclear. miR-26a has demonstrated antifibrotic effects, particularly when its expression is suppressed in ...
Antibacterial activities of novel peptide nucleic acids targeting Salmonella penicillin-binding proteins [0.03%]
针对沙门氏菌青霉素结合蛋白的新肽核酸抗菌活性研究
Mohamed El-Fateh,Charles Viau,Nada Ahmed et al.
Mohamed El-Fateh et al.
Salmonella is a significant global enteric pathogen with a high incidence of multidrug resistance (MDR), which limits therapeutic options and necessitates the development of novel treatments. Antisense peptide nucleic acids (PNAs), which ta...