Harnessing the potential of lipid nanoparticles for the delivery of chemically modified siRNA to combat hepatic adenovirus infection [0.03%]
利用脂质纳米颗粒递送化学修饰的siRNA对抗肝腺病毒感染的潜力研究
Quazi T H Shubhra,A K M Moshiul Alam
Quazi T H Shubhra
Fluorescent nanodiamonds as innovative delivery systems for MiR-34a replacement in breast cancer [0.03%]
荧光纳米钻石作为创新递送系统用于乳腺癌中MiR-34a替代疗法
Marianna Abate,Angela Lombardi,Amalia Luce et al.
Marianna Abate et al.
Nanodiamonds are innovative nanocrystalline carbon particles able to deliver chemically conjugated miRNAs. In oncology, the use of miRNA-based therapies may represent an advantage, based on their ability to simultaneously target multiple in...
Nanoblades allow high-level genome editing in murine and human organoids [0.03%]
纳米刀可在类器官中进行高水平基因组编辑
Victor Tiroille,Adrien Krug,Emma Bokobza et al.
Victor Tiroille et al.
Genome engineering has become more accessible thanks to the CRISPR-Cas9 gene-editing system. However, using this technology in synthetic organs called "organoids" is still very inefficient. This is due to the delivery methods for the CRISPR...
Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C [0.03%]
Lentiviral 基因治疗逆转了伯纳德-索维综合征C型的GPIX表达和表型
Gonzalo Martinez-Navajas,Jorge Ceron-Hernandez,Iris Simon et al.
Gonzalo Martinez-Navajas et al.
Bernard-Soulier syndrome (BSS) is a rare congenital disease characterized by macrothrombocytopenia and frequent bleeding. It is caused by pathogenic variants in three genes (GP1BA, GP1BB, or GP9) that encode for the GPIbα, GPIbβ, and GPIX...
Molnupiravir's mechanism of action drives "error catastrophe" in SARS-CoV-2: A therapeutic strategy that leads to lethal mutagenesis of the virus [0.03%]
莫努匹韦的作用机制使SARS-CoV-2产生“致命错误”：一种导致病毒发生致死性突变的治疗策略
Srijan Chatterjee,Manojit Bhattacharya,Kuldeep Dhama et al.
Srijan Chatterjee et al.
Estrogen-regulated miRs in bone enhance osteoblast differentiation and matrix mineralization [0.03%]
雌激素调控的骨质疏松microRNA促进成骨细胞分化和基质矿化
Michael J Emch,Zofia Wicik,Kirsten G M Aspros et al.
Michael J Emch et al.
Estrogen signaling is critical for the development and maintenance of healthy bone, and age-related decline in estrogen levels contributes to the development of post-menopausal osteoporosis. Most bones consist of a dense cortical shell and ...
Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach [0.03%]
用于模拟外显子跳跃治疗策略的基因模型小鼠中的CLN3巴顿病缓慢进展
Jessica L Centa,Matthew P Stratton,Melissa A Pratt et al.
Jessica L Centa et al.
Genetic mutations that disrupt open reading frames and cause translation termination are frequent causes of human disease and are difficult to treat due to protein truncation and mRNA degradation by nonsense-mediated decay, leaving few opti...
Engineering B cells with customized therapeutic responses using a synthetic circuit [0.03%]
利用合成线路改造B细胞实现特异性治疗性免疫响应
Audrey Page,Marie Delles,Didier Nègre et al.
Audrey Page et al.
The expansion of genetic engineering has brought a new dimension for synthetic immunology. Immune cells are perfect candidates because of their ability to patrol the body, interact with many cell types, proliferate upon activation, and diff...
AAV-vectored base editor trans-splicing delivers dystrophin repair [0.03%]
基于腺相关病毒载体的碱基编辑器介导的反式剪接修复Dystrophin基因突变
Zhen Li,Manuel A F V Gonçalves
Zhen Li
Liver lobe-specific hydrodynamic gene delivery to baboons: A preclinical trial for hemophilia gene therapy [0.03%]
肝叶特异性液动力基因传递给狒狒：血友病基因治疗的临床前试验
Kenya Kamimura,Tsutomu Kanefuji,Takeshi Suda et al.
Kenya Kamimura et al.
Hydrodynamics-based gene transfer has been successfully employed for in vivo gene delivery to the liver of small animals by tail vein injection and of large animals using a computer-assisted and image-guided protocol. In an effort to develo...