Precision gene editing: The power of CRISPR-Cas in modern genetics [0.03%]
精准基因编辑：CRISPR-Cas在现代遗传学中的力量
Jeong H Joo,Soogene Lee,Keun P Kim
Jeong H Joo
Gene editing has transformed molecular biology by enabling precise modifications to genomic DNA across a wide variety of organisms. Gene editing technologies make it possible to add, remove, or modify specific DNA sequences, with a range of...
Prime editing corrects the dilated cardiomyopathy causing RBM20-P633L-mutation in human cardiomyocytes [0.03%]
针对人类心肌细胞扩张型心肌病致病突变RBM20-P633L的prime编辑纠正技术
Alexandra Roman,Anja Zimmer,Michael Gotthardt et al.
Alexandra Roman et al.
Prime editing (PE) is an innovative next-generation gene editing tool that has therapeutic potential in post-mitotic organs, such as the human heart. However, its applicability and efficiency in non-proliferating cells, e.g., human cardiomy...
mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment [0.03%]
变革医药的信使核糖核酸治疗技术：设计、输送和疾病治疗领域的创新
Mark Akinola Ige,Xiaohu Ren,Yang Yang et al.
Mark Akinola Ige et al.
mRNA therapeutics have revolutionized medicine, offering a versatile platform to address previously untreatable diseases. Recent advancements in biotechnology have enabled the efficient production of functional proteins, antibodies, and pep...
mRNA vaccine expressing enterovirus D68 virus-like particles induces potent neutralizing antibodies and protects against infection [0.03%]
表达肠病毒D68体外颗粒的mRNA疫苗可诱导强烈的中和抗体并防止感染
Yuta Kunishima,Kota Senpuku,Chikako Kataoka-Nakamura et al.
Yuta Kunishima et al.
Enterovirus D68 (EV-D68) causes respiratory illness in children. It also causes severe paralysis called acute flaccid myelitis (AFM), which has become a global health threat. Here, we generated an mRNA vaccine expressing virus-like particle...
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa [0.03%]
新型eRF3a降解剂可增强庆大霉素诱导的棘皮松散症无义突变读码框移读通能力
Kathleen L Miao,Brandon Levian,Yingping Hou et al.
Kathleen L Miao et al.
Recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB) are severe blistering skin disorders caused by mutations in genes encoding type VII collagen (COL7A1) and laminin 332 (LAMA3, LAMB3, or LAMC2), res...
Targeting AASS alleviates neurotoxicity and improves mitochondrial function in astrocyte models for pyridoxine-dependent epilepsy [0.03%]
靶向AASS可缓解依赖维生素B6的癫痫的小胶质细胞源性神经毒性及改善线粒体功能
Imke M E Schuurmans,Udo Engelke,Muna Abedrabbo et al.
Imke M E Schuurmans et al.
Pyridoxine-dependent epilepsy (PDE) is a rare neurometabolic disorder of lysine catabolism caused by bi-allelic variants in ALDH7A1. This enzyme deficiency leads to accumulation of neurotoxic metabolites, pyridoxal-phosphate inactivation, a...
Pan-cancer neural regulation pattern with implications for patient stratification and immunotherapy [0.03%]
泛癌神经调节模式及其对患者分层和免疫治疗的启示
Yueying Gao,Jiyu Guo,Wenyi Yang et al.
Yueying Gao et al.
The interplay between the nervous system and cancer plays a central role in regulating oncogenesis. We depict the pan-cancer landscape of neuro-tumor interactions by integration of >10,000 The Cancer Genome Atlas (TCGA) bulk and 239 pan-can...
Selection of specific and efficient siRNAs in new cellular model for Hutchinson-Gilford progeria syndrome therapy [0.03%]
选择特异高效的siRNA用于治疗 Hutchinson-Gilford 前列衰老人综合征的新细胞模型中
Volha Dzianisava,Katarzyna Piekarowicz,Magdalena Machowska et al.
Volha Dzianisava et al.
Hutchinson-Gilford progeria syndrome is a fatal genetic disorder caused by a point mutation in the gene encoding the nuclear envelope protein lamin A/C. The most frequent mutation leads to the synthesis of a shorter version of lamin A named...
Co-delivering a cGAS agonist and antigen via LNPs potentiates cancer immunotherapy [0.03%]
通过LNPs共同递送cGAS激动剂和抗原可增强癌症免疫治疗
Zhaoming Wu,Miao Zhang,Qianqian Ni
Zhaoming Wu
Imogen R Brooks,Carina Graham,Aidin Kazemizadeh et al.
Imogen R Brooks et al.
Prime editing (PE) is a recent advancement in CRISPR-Cas9 technology that involves the fusion of a reverse transcriptase (RT) to a Cas9 nickase (nCas9). This fusion protein is complexed with a prime editing guide RNA (pegRNA), which include...