Cellular uptake of modified mRNA occurs via caveolae-mediated endocytosis, yielding high protein expression in slow-dividing cells [0.03%]
经由网格蛋白包被小坑介导的内吞作用摄取改良型信使RNA，从而在分裂较慢的细胞中获得高效蛋白质表达
Claudia Del Toro Runzer,Shivesh Anand,Carlos Mota et al.
Claudia Del Toro Runzer et al.
Nucleic acids have clear clinical potential for gene therapy. Plasmid DNA (pDNA) was the first nucleic acid to be pursued as a therapeutic molecule. Recently, mRNA came into play as it offers improved safety and affordability. In this study...
Rationale and strategies for the development of safe and effective optimized AAV vectors for human gene therapy [0.03%]
开发用于人类基因治疗的安全有效AAV载体的理据和策略
Arun Srivastava
Arun Srivastava
Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in 332 phase I/II/III clinical trials in a number of human diseases, and in some cases, remarkable clinical efficacy has also been achieved. There are now ...
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy [0.03%]
多条道路通罗马：寡核苷酸疗法多样性助力肌萎缩性脊髓侧索硬化症治疗
Agnieszka Fiszer
Agnieszka Fiszer
Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells [0.03%]
利用CRISPRi特异性靶向DMPK启动子逆转杜氏肌营养不良1型患者肌肉细胞的缺陷
Florent Porquet,Lin Weidong,Kévin Jehasse et al.
Florent Porquet et al.
Myotonic dystrophy type 1 (DM1) is a neuromuscular disease that originates from an expansion of CTG microsatellites in the 3' untranslated region of the DMPK gene, thus leading to the expression of transcripts containing expanded CUG repeat...
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice [0.03%]
用于DMD小鼠的tricyclo-DNA ASO可恢复大脑中的dystrophin蛋白并改善神经行为缺陷
Muthukumar Karuppasamy,Matthew S Alexander
Muthukumar Karuppasamy
M Elena Martin
M Elena Martin
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease [0.03%]
用于治疗斯特格病变的诱导多能干细胞中的CRISPR-Cas9：个性化治疗新时代
Soujanya Kuntam,Pradeep Reddy Cingaram
Soujanya Kuntam
Landscape and perturbation of enhancer-driven core transcription regulatory circuits in cancer [0.03%]
癌症中增强子驱动的核心转录调控回路的景观和扰动
Juan Xu,Tiantongfei Jiang,Jiyu Guo et al.
Juan Xu et al.
Role of miR-300-3p in Leydig cell function and differentiation: A therapeutic target for obesity-related testosterone deficiency [0.03%]
miR-300-3p在曲细精管间质细胞功能及分化中的作用及其作为肥胖相关性睾酮缺乏症治疗靶点的潜力
Jinlian Liang,Derong Chen,Ziyan Xiao et al.
Jinlian Liang et al.
MicroRNAs (miRNAs) regulate various cellular functions, but their specific roles in the regulation of Leydig cells (LCs) have yet to be fully understood. Here, we found that the expression of miR-300-3p varied significantly during the diffe...
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity [0.03%]
巨噬细胞靶向RNA传递调节巨噬细胞极化 Oversized脂质体促进巨噬细胞靶向RNA传递以调节巨噬细胞极化
Dongqing Wang,Jiayu Zhang,Jun Chen
Dongqing Wang