Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors [0.03%]
利用下一代纳米质粒载体改进细胞和基因疗法的安全性和性能
James A Williams,Patrick A Paez
James A Williams
The cell and gene therapy industry has employed the same plasmid technology for decades in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA production. While canonical plasmids contain antibiotic resistance ...
DNAJB6 isoform specific knockdown: Therapeutic potential for limb girdle muscular dystrophy D1 [0.03%]
DNAJB6异构体特异性敲低：治疗肢带型肌营养不良D1的潜力
Andrew R Findlay,May M Paing,Jil A Daw et al.
Andrew R Findlay et al.
Dominant missense mutations in DNAJB6, a co-chaperone of HSP70, cause limb girdle muscular dystrophy (LGMD) D1. No treatments are currently available. Two isoforms exist, DNAJB6a and DNAJB6b, each with distinct localizations in muscle. Muta...
Lipid nanoparticle-encapsulated, chemically modified anti-adenoviral siRNAs inhibit hepatic adenovirus infection in immunosuppressed Syrian hamsters [0.03%]
免疫抑制叙利亚仓鼠肝腺病毒感染的脂质纳米颗粒包封化学修饰抗腺病毒siRNA抑制作用
Anja Geisler,Babette Dieringer,Leslie Elsner et al.
Anja Geisler et al.
RNA interference has demonstrated its potential as an antiviral therapy for treatment of human adenovirus (hAd) infections. The only existing viral vector-based system for delivery of anti-adenoviral artificial microRNAs available for in vi...
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection [0.03%]
脂质纳米颗粒传递限制了脑室内注射后反义寡核苷酸的活性和细胞分布
Amy E Byrnes,Sara L Dominguez,Chun-Wan Yen et al.
Amy E Byrnes et al.
Antisense oligonucleotide (ASO) therapeutics are being investigated for a broad range of neurological diseases. While ASOs have been effective in the clinic, improving productive ASO internalization into target cells remains a key area of f...
MutSα and MutSβ as size-dependent cellular determinants for prime editing in human embryonic stem cells [0.03%]
人类胚胎干细胞中基于大小的细胞决定因子MutSα和MutSβ在碱基编辑中的作用
Ju-Chan Park,Yun-Jeong Kim,Jun Hee Han et al.
Ju-Chan Park et al.
Precise genome editing in human pluripotent stem cells (hPSCs) has potential applications in isogenic disease modeling and ex vivo stem cell therapy, necessitating diverse genome editing tools. However, unlike differentiated somatic cells, ...
Strategic self-limiting production of infectious HIV particles by CRISPR in permissive cells [0.03%]
在允许细胞中利用CRISPR技术策略性地限制生产感染性HIV颗粒
Hong Liu,Chen Chen,Shuren Liao et al.
Hong Liu et al.
Post-translational glycosylation of the HIV-1 envelope protein involving precursor glycan trimming by mannosyl oligosaccharide glucosidase (MOGS) is critically important for morphogenesis of virions and viral entry. Strategic editing of the...
Targeting microRNA-10 in glioma; a focus with potential therapeutic application in genome editing [0.03%]
靶向胶质瘤中的microRNA-10；基因组编辑中具有潜在治疗应用的研究重点
Katharina Maus,Felix Jansen,Mohammed Rabiul Hosen
Katharina Maus
The impact of nucleoside base modification in mRNA vaccine is influenced by the chemistry of its lipid nanoparticle delivery system [0.03%]
mRNA疫苗的碱基修饰效果受脂质纳米粒传递系统化学性质影响
Marie-Clotilde Bernard,Emilie Bazin,Nadine Petiot et al.
Marie-Clotilde Bernard et al.
The use of modified nucleosides is an important approach to mitigate the intrinsic immunostimulatory activity of exogenous mRNA and to increase its translation for mRNA therapeutic applications. However, for vaccine applications, the intrin...
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling [0.03%]
miR-409-3p缺乏通过调节DNAJB9/p38MAPK信号改善心肌新生血管和功能
Furkan Bestepe,Colette Fritsche,Kartik Lakhotiya et al.
Furkan Bestepe et al.
Angiogenesis is critical for tissue repair following myocardial infarction (MI), which is exacerbated under insulin resistance or diabetes. MicroRNAs are regulators of angiogenesis. We examined the metabolic regulation of miR-409-3p in post...
A protein domain-oriented approach to expand the opportunities of therapeutic exon skipping for USH2A-associated retinitis pigmentosa [0.03%]
一种蛋白质结构域导向的方法，用于扩展USH2A相关视网膜色素变性的治疗性外显子跳读的机遇
Renske T W Schellens,Sanne Broekman,Theo Peters et al.
Renske T W Schellens et al.
Loss-of-function mutations in USH2A are among the most common causes of syndromic and non-syndromic retinitis pigmentosa (RP). We previously presented skipping of USH2A exon 13 as a promising treatment paradigm for USH2A-associated RP. Howe...