Katye M Fichter,Tahereh Setayesh,Punam Malik
Katye M Fichter
CRISPR-Cas technologies have the potential to revolutionize genetic medicine. However, work is still needed to make this technology clinically efficient for gene correction. A barrier to making precise genetic edits in the human genome is c...
A review from biological mapping to computation-based subcellular localization [0.03%]
从生物制图到基于计算的细胞内定位综述
Jing Li,Quan Zou,Lei Yuan
Jing Li
Subcellular localization is crucial to the study of virus and diseases. Specifically, research on protein subcellular localization can help identify clues between virus and host cells that can aid in the design of targeted drugs. Research o...
Retraction Notice to: MicroRNA-494 Inhibits the LRG1 Expression to Induce Proliferation and Migration of VECs in Rats following Myocardial Infarction [0.03%]
撤稿：MicroRNA-494通过抑制LRG1表达诱导心肌梗死后VEC增殖和迁移的机制研究收稿通知
Qiang Su,Xiang-Wei Lv,Yu-Han Sun et al.
Qiang Su et al.
[This retracts the article DOI: 10.1016/j.omtn.2019.08.007.]. © 2023 The Author(s).
Retraction of Publication
Molecular therapy. Nucleic acids. 2023 May 11:32:667. DOI:10.1016/j.omtn.2023.05.006 2023
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition [0.03%]
一个条件性的RNA聚合酶II单启动子驱动HIV诱导的、CRISPR介导的环咽蛋白T1抑制和抗HIV作用
Srinivasan Chinnapaiyan,Maria-Jose Santiago,Kingshuk Panda et al.
Srinivasan Chinnapaiyan et al.
Gene editing using clustered regularly interspaced short palindromic repeats (CRISPR) targeted to HIV proviral DNA has shown excision of HIV from infected cells. However, CRISPR-based HIV excision is vulnerable to viral escape. Targeting ce...
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations [0.03%]
用于常见杜氏肌营养不良症突变矫正的单交换编辑
Andreas C Chai,Francesco Chemello,Hui Li et al.
Andreas C Chai et al.
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive muscle weakness and wasting caused by the absence of dystrophin protein. Current gene therapy approaches using antisense oligonucleotides require lifelon...
LncRNA ZNF593-AS alleviates diabetic cardiomyopathy via suppressing IRF3 signaling pathway [0.03%]
长链非编码RNA ZNF593-AS通过抑制IRF3信号通路缓解糖尿病心肌病
Rong Xie,Jiahui Fan,Jianpei Wen et al.
Rong Xie et al.
Diabetes could directly induce cardiac injury, leading to cardiomyopathy. However, treatment strategies for diabetic cardiomyopathy remain limited. ZNF593-AS knockout and cardiomyocyte-specific transgenic mice were constructed. In addition,...
Streamlined DNA-encoded small molecule library screening and validation for the discovery of novel chemotypes targeting BET proteins [0.03%]
用于发现针对BET蛋白的新化学亚型的DNA编码的小分子文库筛选和验证的新方法
Seoyeon Jeong,Hwa-Ryeon Kim,June-Ha Shin et al.
Seoyeon Jeong et al.
Targeting aberrant epigenetic programs that drive tumorigenesis is a promising approach to cancer therapy. DNA-encoded library (DEL) screening is a core platform technology increasingly used to identify drugs that bind to protein targets. H...
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1 [0.03%]
阻断还是降解？针对DM1中三核苷酸重复扩增转录本的反义策略的在靶和脱靶效应之间的权衡
Najoua El Boujnouni,M Leontien van der Bent,Marieke Willemse et al.
Najoua El Boujnouni et al.
Antisense oligonucleotide (ASO) therapies for myotonic dystrophy type 1 (DM1) are based on elimination of transcripts containing an expanded repeat or inhibition of sequestration of RNA-binding proteins. This activity is achievable by both ...
In utero delivery of miRNA induces epigenetic alterations and corrects pulmonary pathology in congenital diaphragmatic hernia [0.03%]
宫内递送miRNA诱导表观遗传改变并能矫正先天性膈疝肺病理损伤
Sarah J Ullrich,Nicholas K Yung,Tory J Bauer-Pisani et al.
Sarah J Ullrich et al.
Structural fetal diseases, such as congenital diaphragmatic hernia (CDH) can be diagnosed prenatally. Neonates with CDH are healthy in utero as gas exchange is managed by the placenta, but impaired lung function results in critical illness ...
Poly(β-amino ester)s-based nanovehicles: Structural regulation and gene delivery [0.03%]
基于聚(β-氨基酯)的纳米载体：结构调控与基因递送
Jiayu Zhang,Xiaomeng Cai,Rui Dou et al.
Jiayu Zhang et al.
The first poly(β-amino) esters (PβAEs) were synthesized more than 40 years ago. Since 2000, PβAEs have been found to have excellent biocompatibility and the capability of ferrying gene molecules. Moreover, the synthesis process of PβAEs...