Improved cross-protection and immunity against influenza A virus in mice using a novel mRNA vaccine with optimized design of RNA sequence [0.03%]
利用优化的RNA序列设计新型mRNA疫苗改善小鼠对甲型流感病毒的交叉保护和免疫性
Tangqi Wang,Ruiwen Han,Zhanyihao Hao et al.
Tangqi Wang et al.
Conventional influenza vaccines provide strain-specific immunity, and their production can be affected by egg-culture-adapted mutations. A universal influenza vaccine with conserved antigens and novel vaccine platforms is urgently required....
CRISPR-Cas editing technologies for viral-mediated gene therapies of human diseases: Mechanisms, progress, and challenges [0.03%]
基于CRISPR-Cas的病毒介导的人类疾病基因治疗技术：机制、进展和挑战
Boris Kantor,Leanne Duke,Pradeep G Bhide
Boris Kantor
The gene therapy landscape has evolved substantially in recent years, beginning with the approval of the first adeno-associated virus-based gene therapy, Luxterna, in 2017. Since then, the US FDA has approved nearly 30 new viral gene therap...
Synergic microRNAs suppress human glioblastoma progression by modulating clinically relevant targets [0.03%]
协同微小核糖核酸通过调节临床相关目标抑制人类胶质母细胞瘤进展
Silvia Rancati,Rui C Pereira,Michele Schlich et al.
Silvia Rancati et al.
Glioblastoma (GBM) is a highly aggressive brain tumor characterized by therapy-resistant glioma stem-like cells (GSCs) and extensive infiltration into surrounding brain tissue. MicroRNAs (miRNAs) are pleiotropic post-transcriptional regulat...
Development of a novel-ionizable-lipid-based mRNA vaccine for broad protection against Streptococcus pneumoniae [0.03%]
开发一种新型可离子化脂质基mRNA疫苗以对抗肺炎链球菌广泛感染
Shi Xu,Guoqing Qi,Rui Liu et al.
Shi Xu et al.
Recently, messenger RNA (mRNA)-based vaccine technology has made significant advances in preventing pathogenic microbial infections. The composition and physicochemical properties of lipid nanoparticle (LNP) determine the delivery efficienc...
Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections [0.03%]
反复注射反义寡核苷酸后mdx23营养不良小鼠的行为学改善现象
Artadokht Aghaeipour,Manuela Mitsogiannis,Claire Fergus et al.
Artadokht Aghaeipour et al.
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by mutations in the DMD gene that disrupt the production of functional dystrophin proteins. Intellectual disability and neurobehavioral complications including auti...
Toward designer magic bullets: Epigenetic editing to empower CAR T cells [0.03%]
朝向设计魔弹：表观遗传学编辑增强CAR-T细胞功能
Laura Horvathova,Valerie R Wiersma,Marianne G Rots
Laura Horvathova
Erratum: Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency [0.03%]
Correction: 合理设计用于PKU和SSADH缺乏症的哌嗪和环己烷离子化脂质
Jiyeon Son,Hyunjung Yoo,Joonyoung Park et al.
Jiyeon Son et al.
[This corrects the article DOI: 10.1016/j.omtn.2025.102697.]. © 2025 The Author(s).
Published Erratum
Molecular therapy. Nucleic acids. 2025 Nov 7;36(4):102735. DOI:10.1016/j.omtn.2025.102735 2025
Jesse H Erasmus
Jesse H Erasmus
Fishing for novel HDAC inhibitor compounds to treat Duchenne muscular dystrophy [0.03%]
寻找新颖的HDAC抑制剂化合物以治疗杜氏肌营养不良症
Katherine G English,Matthew S Alexander
Katherine G English
An siRMSD parameter of structural distortion induced by chemical modification is predictive of the off-target effect of siRNA [0.03%]
由化学修饰诱发的siRNA结构扭曲的siRMSD参数可预测其脱靶效应大小
Seongjin An,Kohei Nomura,Yoshiaki Kobayashi et al.
Seongjin An et al.
We developed siRMSD, a predictive parameter for off-target effects induced by chemical modifications, to optimize siRNA therapeutics. In RNA interference, small interfering RNA (siRNA) suppresses gene function by degrading mRNA with perfect...