Mini-PE, a prime editor with compact Cas9 and truncated reverse transcriptase [0.03%]
微型PE编辑器：紧凑型Cas9和截短的逆转录酶
Ting Lan,Huangyao Chen,Chengcheng Tang et al.
Ting Lan et al.
Prime editor (PE) is a versatile genome editing tool that does not need extra DNA donors or inducing double-strand breaks. However, in vivo implementation of PE remains a challenge because of its oversized composition. In this study, we scr...
Novel hybrid silicon-lipid nanoparticles deliver a siRNA to cure autosomal dominant osteopetrosis in mice. Implications for gene therapy in humans [0.03%]
新型硅脂质体纳米颗粒将siRNA递送至小鼠以治疗常染色体显性遗传性成骨不全症及对人类基因治疗的意义
Antonio Maurizi,Piergiorgio Patrizii,Anna Teti et al.
Antonio Maurizi et al.
Rare skeletal diseases are still in need of proper clinically available transfection agents as the major challenge for first-in-human translation relates to intrinsic difficulty in targeting bone without exacerbating any inherent toxicity d...
The circular RNA Ataxia Telangiectasia Mutated regulates oxidative stress in smooth muscle cells in expanding abdominal aortic aneurysms [0.03%]
ATM circular RNA调节扩张型腹主动脉瘤平滑肌细胞内的氧化应激反应
Francesca Fasolo,Greg Winski,Zhaolong Li et al.
Francesca Fasolo et al.
An abdominal aortic aneurysm (AAA) is a pathological widening of the aortic wall characterized by loss of smooth muscle cells (SMCs), extracellular matrix degradation, and local inflammation. This condition is often asymptomatic until ruptu...
PINE-TREE enables efficient enrichment of prime-edited hPSCs [0.03%]
高效的PINE-TREE方法 enriches prime-edited hPSCs
Mary S Jia,Christopher E Nelson
Mary S Jia
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture [0.03%]
电穿孔导入抗微小核糖核酸抑制二维和三维上皮细胞培养中微小核糖核酸-34a的水平
Julie Weidner,Ewa Kolosionek,Reetta Holmila et al.
Julie Weidner et al.
MicroRNAs are attractive therapeutic targets in many diseases, including chronic obstructive pulmonary disease and idiopathic pulmonary fibrosis. Among microRNA inhibitors antimiRs have been proven successful in lowering aberrant microRNA l...
Christina Huang,Jack L Arbiser
Christina Huang
Cell-type-specific alternative polyadenylation promotes oncogenic gene expression in non-small cell lung cancer progression [0.03%]
非小细胞肺癌发生发展过程中细胞类型特异性选择性多腺苷酸化促进致癌基因表达
Kexin Huang,Yun Zhang,Xiaorui Shi et al.
Kexin Huang et al.
Disrupted alternative polyadenylation (APA) is frequently involved in tumorigenesis and cancer progression by regulating the gene expression of oncogenes and tumor suppressors. However, limited knowledge of tumor-type- and cell-type-specifi...
Adam J Pollak,Luyi Zhao,Stanley T Crooke
Adam J Pollak
Single-stranded phosphorothioate oligonucleotides (PS-oligos) can activate TLR9, leading to an innate immune response. This can occur with PS-oligos containing unmethylated CpG sites, the canonical motif, or PS-oligos that do not contain th...
Putting a stop to nonsense: Revisiting gene correction therapy for neurofibromatosis type 1 [0.03%]
终结糊涂事——重新审视NF1基因修正疗法
David H Gutmann
David H Gutmann
Selection of a novel cell-internalizing RNA aptamer specific for CD22 antigen in B cell acute lymphoblastic leukemia [0.03%]
选择针对B细胞急性淋巴细胞白血病CD22抗原的新型细胞内化RNA适配体
Dario Ruiz-Ciancio,Li-Hsien Lin,Suresh Veeramani et al.
Dario Ruiz-Ciancio et al.
Despite improvements in B cell acute lymphoblastic leukemia (B-ALL) treatment, a significant number of patients experience relapse of the disease, resulting in poor prognosis and high mortality. One of the drawbacks of current B-ALL treatme...