Improved tropoelastin synthesis in the skin by codon optimization and nucleotide modification of tropoelastin-encoding synthetic mRNA [0.03%]
通过密码子优化和核苷酸修饰的编码角蛋白原合成mRNA改善皮肤中角蛋白原合成
Sonia Golombek,Thomas Hoffmann,Ludmilla Hann et al.
Sonia Golombek et al.
Loss of elastin due to aging, disease, or injury can lead to impaired tissue function. In this study, de novo tropoelastin (TE) synthesis is investigated in vitro and in vivo using different TE-encoding synthetic mRNA variants after codon o...
Intratumoral immunotherapy with mRNAs encoding chimeric protein constructs encompassing IL-12, CD137 agonists, and TGF-β antagonists [0.03%]
包含IL-12、CD137激动剂和TGF-β拮抗剂嵌合蛋白构建物的mRNA瘤内免疫治疗
Assunta Cirella,Elixabet Bolaños,Carlos Luri-Rey et al.
Assunta Cirella et al.
Intratumoral immunotherapy strategies for cancer based on interleukin-12 (IL-12)-encoding cDNA and mRNA are under clinical development in combination with anti-PD-(L)1 monoclonal antibodies. To make the most of these approaches, we have con...
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis- regulation in human and mouse [0.03%]
Cis-Cardio：一个人类和小鼠心血管相关顺式调控的综合分析平台
Chao Song,Yuexin Zhang,Hong Huang et al.
Chao Song et al.
Cis-regulatory elements are important molecular switches in controlling gene expression and are regarded as determinant hubs in the transcriptional regulatory network. Collection and processing of large-scale cis-regulatory data are urgent ...
Long-term muscle-specific overexpression of DOK7 in mice using AAV9-tMCK-DOK7 [0.03%]
使用AAV9-tMCK-DOK7在小鼠中长期肌肉特异性过表达DOK7蛋白质
Yu-Ting Huang,Hannah R Crick,Helena Chaytow et al.
Yu-Ting Huang et al.
Neuromuscular junction (NMJ) dysfunction underlies several diseases, including congenital myasthenic syndromes (CMSs) and motor neuron disease (MND). Molecular pathways governing NMJ stability are therefore of interest from both biological ...
Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors [0.03%]
利用碱基编辑器通过跳跃DMD外显子45治疗杜氏肌营养不良症
Michael Gapinske,Jackson Winter,Devyani Swami et al.
Michael Gapinske et al.
Duchenne muscular dystrophy is an X-linked monogenic disease caused by mutations in the dystrophin gene (DMD) characterized by progressive muscle weakness, leading to loss of ambulation and decreased life expectancy. Since the current stand...
Efficient circular RNA engineering by end-to-end self-targeting and splicing reaction using Tetrahymena group I intron ribozyme [0.03%]
利用四膜虫群I内含子核糖酶端到端自靶标及剪接反应高效工程化环状RNA
Kyung Hyun Lee,Seongcheol Kim,Jaehwi Song et al.
Kyung Hyun Lee et al.
Circular RNA (circRNA) has various advantages over linear mRNA that is gaining success as a new vaccine and therapeutic agent. Thus, circRNA and its engineering methods have attracted attention recently. In this study, we developed a new in...
Intratumoral injection of IL-12-encoding mRNA targeted to CSFR1 and PD-L1 exerts potent anti-tumor effects without substantial systemic exposure [0.03%]
靶向CSF1R和PD-L1的编码IL-12的mRNA瘤内注射可产生显著的抗肿瘤效果，且没有严重的全身暴露风险
Claudia Augusta Di Trani,Assunta Cirella,Leire Arrizabalaga et al.
Claudia Augusta Di Trani et al.
IL-12 is a potent cytokine for cancer immunotherapy. However, its systemic delivery as a recombinant protein has shown unacceptable toxicity in the clinic. Currently, the intratumoral injection of IL-12-encoding mRNA or DNA to avoid such si...
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles [0.03%]
携带EGFR适配体的多功能RNA纳米颗粒靶向非小细胞肺癌中致癌KRAS基因治疗研究
Linlin Yang,Zhefeng Li,Daniel W Binzel et al.
Linlin Yang et al.
KRAS mutations are one of the most common oncogenic driver mutations in human cancers, including non-small cell lung cancer (NSCLC), and have established roles in cancer pathogenesis and therapeutic resistance. The development of effective ...
Treatment of autosomal dominant retinitis pigmentosa caused by RHO-P23H mutation with high-fidelity Cas13X in mice [0.03%]
在小鼠中使用高保真Cas13X治疗由RHO-P23H突变引起的常染色体显性视网膜色素变性
Zixiang Yan,Yuqin Yao,Luyao Li et al.
Zixiang Yan et al.
Mutations in Rhodopsin (RHO) gene commonly cause autosomal dominant retinitis pigmentosa (adRP) without effective therapeutic treatment so far. Compared with genomic DNA-targeting CRISPR-Cas9 system, Cas13 edits RNA for therapeutic applicat...
A neutralizable dimeric anti-thrombin aptamer with potent anticoagulant activity in mice [0.03%]
一种具有强抗凝血活性的小鼠双链型中和端 thrombin 配体类抗体
Masanobu Nagano,Kazuki Kubota,Asuka Sakata et al.
Masanobu Nagano et al.
Heparin-induced thrombocytopenia (HIT) is a complication caused by administration of the anticoagulant heparin. Although the number of patients with HIT has drastically increased because of coronavirus disease 2019 (COVID-19), the currently...