Development of new non-viral systems for genetic modification of senescent cells [0.03%]
新型非病毒系统用于基因编辑衰老细胞的发展研究
Junquera López-Seijas,Diego Miranda-Balbuena,Alba Iglesias-Fente et al.
Junquera López-Seijas et al.
Senescence is a process characterized by a prolonged irreversible cell-cycle arrest. The accumulation of senescent cells in tissues is related to aging and to the development of age-related diseases. Recently, gene therapy has emerged as a ...
GOLGA8 increases bulk antisense oligonucleotide uptake and activity in mammalian cells [0.03%]
GOLGA8增加哺乳动物细胞中Bulk反义寡核苷酸的摄取和活性
Moira A McMahon,Meghdad Rahdar,Swagatam Mukhopadhyay et al.
Moira A McMahon et al.
Antisense oligonucleotides (ASOs) are short synthetic nucleic acids that recognize and bind to complementary RNA to modulate gene expression. It is well established that single-stranded, phosphorothioate-modified ASOs enter cells independen...
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies [0.03%]
结合基因添加和基因沉默策略的治疗镰状细胞疾病的新型慢病毒载体
Mégane Brusson,Anne Chalumeau,Pierre Martinucci et al.
Mégane Brusson et al.
Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α2βS 2). Transplantation of autologous hematopoietic stem and progenitor cells (HSPCs) transduced with lentiviral...
Development of DNA aptamers for visualization of glial brain tumors and detection of circulating tumor cells [0.03%]
DNA适配体的发展以实现胶质脑肿瘤的可视化和循环肿瘤细胞的检测
Anna S Kichkailo,Andrey A Narodov,Maria A Komarova et al.
Anna S Kichkailo et al.
Here, we present DNA aptamers capable of specific binding to glial tumor cells in vitro, ex vivo, and in vivo for visualization diagnostics of central nervous system tumors. We selected the aptamers binding specifically to the postoperative...
Circulating extracellular vesicles promote recovery in a preclinical model of intracerebral hemorrhage [0.03%]
循环细胞外囊泡促进脑内出血的恢复：基于动物模型的研究
Fernando Laso-García,Laura Casado-Fernández,Dolores Piniella et al.
Fernando Laso-García et al.
Circulating extracellular vesicles (EVs) are proposed to participate in enhancing pathways of recovery after stroke through paracrine signaling. To verify this hypothesis in a proof-of-concept study, blood-derived allogenic EVs from rats an...
Self-transfecting GMO-PMO chimera targeting Nanog enable gene silencing in vitro and suppresses tumor growth in 4T1 allografts in mouse [0.03%]
一种新型的以Nanog为靶点的基因沉默嵌合体自我感染载体及其应用
Ujjal Das,Jayanta Kundu,Pallab Shaw et al.
Ujjal Das et al.
Phosphorodiamidate morpholino oligonucleotide (PMO)-based antisense reagents cannot enter cells without the help of a delivery technique, which limits their clinical applications. To overcome this problem, self-transfecting guanidinium-link...
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice [0.03%]
三环形DNA反义寡核苷酸部分恢复脑内dystrophin可缓解mdx52小鼠情感障碍症状
Amel Saoudi,Sacha Barberat,Olivier le Coz et al.
Amel Saoudi et al.
The mdx52 mouse model recapitulates a frequent mutation profile associated with brain involvement in Duchenne muscular dystrophy. Deletion of exon 52 impedes expression of two dystrophins (Dp427, Dp140) expressed in brain, and is eligible f...
In vivo genome editing for hemophilia B therapy by the combination of rebalancing and therapeutic gene knockin using a viral and non-viral vector [0.03%]
利用病毒和非病毒载体再平衡及治疗基因敲入的体内基因组编辑用于乙型血友病治疗
Jeong Hyeon Lee,Jeong Pil Han,Dong Woo Song et al.
Jeong Hyeon Lee et al.
Recent therapeutic strategies for hemophilia include long-term therapeutic gene expression using adeno-associated virus (AAV) and rebalancing therapy via the downregulation of anticoagulant pathways. However, these approaches have limitatio...
MiR34 contributes to spinal muscular atrophy and AAV9-mediated delivery of MiR34a ameliorates the motor deficits in SMA mice [0.03%]
MiR34参与脊髓性肌萎缩症且携带MiR34a的AAV9能够改善SMA小鼠的运动障碍
Tai-Heng Chen,Shih-Hsin Chang,Yu-Fu Wu et al.
Tai-Heng Chen et al.
Spinal muscular atrophy (SMA) is a neurodegenerative disease characterized by the selective loss of spinal motor neurons (MNs) and concomitant muscle weakness. Mutation of SMN1 is known to cause SMA, and restoring SMN protein levels via ant...
Resistance to nirmatrelvir due to mutations in the Mpro in the subvariants of SARS-CoV-2 Omicron: Another concern? [0.03%]
由于SARS-CoV-2奥密克戎亚变体中Mpro突变导致对尼马曲韦耐药：另一个需要关注的问题？
Srijan Chatterjee,Manojit Bhattacharya,Kuldeep Dhama et al.
Srijan Chatterjee et al.