Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C [0.03%]
lentiviral基因疗法可恢复Bernard-Soulier综合征C型患者的GPIX表达和表型
Raimondo De Cristofaro
Raimondo De Cristofaro
EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method [0.03%]
剂量、采集介质和分离方法决定的EV介导的肌源性分化的促进作用
Britt Hanson,Ioulia Vorobieva,Wenyi Zheng et al.
Britt Hanson et al.
Extracellular vesicles (EVs) have been implicated in the regulation of myogenic differentiation. C2C12 murine myoblast differentiation was reduced following treatment with GW4869 or heparin (to inhibit exosome biogenesis and EV uptake, resp...
Feng Chen,Qiongwei Ke,Wenyan Wei et al.
Feng Chen et al.
Apolipoprotein E (ApoE) is a multifunctional protein critical for lipid metabolism and cholesterol homeostasis. In addition to being a well known genetic determinant of both neurodegenerative and cardiovascular diseases, ApoE is frequently ...
Unveiling the intricacies of gene delivery: Caveolae-mediated endocytosis induces efficient mRNA delivery in slow-dividing cells [0.03%]
揭秘基因传递的奥秘：胞痕介导的内吞作用可有效将mRNA递送至生长缓慢的细胞中
Sabine den Roover,Joeri L Aerts
Sabine den Roover
Cathepsin B-activatable cyclic antisense oligonucleotides for cell-specific target gene knockdown in vitro and in vivo [0.03%]
一种细胞特异性敲低靶基因的体内和体外环状反义寡核苷酸疗法
Zhongyu Wang,Xinli Fan,Guanqun Mu et al.
Zhongyu Wang et al.
Trigger-activatable antisense oligonucleotides have been widely applied to regulate gene function. Among them, caged cyclic antisense oligonucleotides (cASOs) maintain a specific topology that temporarily inhibits their interaction with tar...
Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene [0.03%]
携带全长人类染色体人工载体的抗肌营养不良基因敲除猪的性状特征研究
Masahito Watanabe,Hitomaru Miyamoto,Kazutoshi Okamoto et al.
Masahito Watanabe et al.
Mammalian artificial chromosomes have enabled the introduction of extremely large amounts of genetic information into animal cells in an autonomously replicating, nonintegrating format. However, the evaluation of human artificial chromosome...
Anna Andrzejewska,Renata Grzela,Anna Stankiewicz-Drogon et al.
Anna Andrzejewska et al.
We previously have shown that mRNA-based engineering may enhance mesenchymal stem cell (MSC) trafficking. However, optimal conditions for in vitro mRNA engineering of MSCs are unknown. Here, we investigated several independent variables: (1...
PINE-TREE enables highly efficient genetic modification of human cell lines [0.03%]
高效的PINE-TREE人类细胞系基因组编辑系统
Carlye Frisch,William W Kostes,Brooke Galyon et al.
Carlye Frisch et al.
Prime editing technologies enable precise genome editing without the caveats of CRISPR nuclease-based methods. Nonetheless, current approaches to identify and isolate prime-edited cell populations are inefficient. Here, we established a flu...
Xavier Bofill-De Ros
Xavier Bofill-De Ros
Ida Juhl Corydon,Bjørn Kristensen Fabian-Jessing,Thomas Stax Jakobsen et al.
Ida Juhl Corydon et al.
The year 2023 marks the 25th anniversary of the discovery of RNAi. RNAi-based therapeutics enable sequence-specific gene knockdown by eliminating target RNA molecules through complementary base-pairing. A systematic review of published and ...