Optimization of siRNA therapeutics targeting MIAT for cardioprotection in myocardial ischemia/reperfusion injury [0.03%]
针对心肌缺血/再灌注损伤的长链非编码RNA MIAT的小干扰RNA递送及优化以实现心脏保护作用
Xiao-Rong Ma,Tong-Meng Yan,Yu Pan et al.
Xiao-Rong Ma et al.
Myocardial ischemia/reperfusion (MI/R) injury is a leading cause of heart failure, and novel therapeutic strategies are urgently needed to improve patient outcomes. Long non-coding RNA (lncRNA) myocardial infarction-associated transcript (M...
SARS-CoV-2 infection in hiPSC-derived neurons is cathepsin-dependent and causes differential accumulation of HIF1ɑ and phosphorylated tau [0.03%]
hiPSC来源的神经元中SARS-CoV-2感染依赖于猫科动物溶酶体蛋白酶并导致HIF1ɑ和磷酸化tau蛋白的不同积累
Pinja Kettunen,Janika Ruuska,Tania Quirin et al.
Pinja Kettunen et al.
The severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has been shown to infect areas of the human brain and a subset of neurons in vitro. We have previously demonstrated that the virus enters human induced pluripotent stem cell (...
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration [0.03%]
比较脑室给药和腰椎腔给药后MALAT1反义寡核苷酸的分布情况
Michelle M Boyd,Curt Mazur,Kaylee Pribnow et al.
Michelle M Boyd et al.
Several antisense oligonucleotide (ASO) therapies are currently in clinical trials or approved for treatment of central nervous system (CNS) diseases. Achieving adequate distribution of systemically delivered ASOs in CNS tissues has been hi...
A novel gene therapy platform for the treatment of type 2 diabetes and obesity [0.03%]
一种用于治疗2型糖尿病和肥胖症的新基因疗法平台
Jared Lourie,Alex Goraltchouk,Judith M Hollander et al.
Jared Lourie et al.
Diabetes and obesity are a growing healthcare and socioeconomic crisis of unprecedented proportions. Driven in part by the westernization of diets around the world, the prevalence of individuals with type 2 diabetes alone is projected to ex...
Seokju Park,Seong-Ho Park,Jin-Seon Oh et al.
Seokju Park et al.
miRNA-mimicking short hairpin RNAs (shRNAmirs), which depend on the endogenous miRNA biogenesis pathway, have been widely used to investigate gene function and to develop therapeutic strategies due to their stable and robust knockdown of ta...
Advancing precision antiviral therapy: The value, challenges, and prospects of a novel antiviral crRNA screening platform [0.03%]
精准抗病毒治疗的推进——新型抗病毒crRNA筛选平台的价值、挑战与前景
Shuaiyin Chen,Jinzhao Long,Guangcai Duan
Shuaiyin Chen
Antisense oligonucleotide-based switches for conditional translation of synthetic mRNA [0.03%]
基于反义寡核苷酸的开关控制合成mRNA的条件性翻译
Francis Combes,Frida J Pettersson,Thanh-Huong Bui
Francis Combes
Synthetic messenger RNA (mRNA) therapeutics requires precise control over their translation to minimize off-target effects. We introduce an antisense oligonucleotide (ASO)-based switch concept that regulates synthetic mRNA translation based...
Subnuclear organization and mislocalization of plasmids reduce transgene expression [0.03%]
质粒的次核型结构及其定位异常可降低基因表达水平
Ningyang Gu,Uday K Baliga,Joseph J Porter et al.
Ningyang Gu et al.
The expression of DNA within the nucleus is controlled by both promoter structure and sequence and higher-order organization of the nucleus, but how these two affect each other has not been greatly studied. We have previously shown that pla...
Prime editing for the investigation of aberrant splicing defect associated with a pathogenic PRPH2 variant [0.03%]
针对致病性PRPH2变异导致的剪接异常的prime编辑研究
Bruna Lopes da Costa,Kyle M Helms,Keith Theodore et al.
Bruna Lopes da Costa et al.
The human Peripherin 2 (PRPH2) gene, essential for the structure and function of photoreceptor outer segments, is implicated in a range of inherited retinal diseases (IRDs). This study focuses on the pathogenic c.828+1G>A PRPH2 splice site ...
Unlocking mRNA-driven CRISPR-Cas9 gene therapy via optimizing mRNA and the delivery vectors [0.03%]
通过优化mRNA和递送载体解锁基于mRNA的CRISPR-Cas9基因治疗
Shengyi Wang,Xiaoyu Xu,Tapani Viitala et al.
Shengyi Wang et al.
mRNA-driven CRISPR-Cas9 gene therapies have garnered widespread attention due to their ability to maintain highly efficient editing activity while preventing integration into the host cell genome. However, the widespread clinical applicatio...