hafoe: an interactive tool for the analysis of chimeric AAV libraries after random mutagenesis [0.03%]
Hafoe：随机诱变后分析嵌合AAV文库的交互式工具
Tatevik Jalatyan,Erik Aznauryan,Rokib Hasan et al.
Tatevik Jalatyan et al.
Naturally occurring adeno-associated viruses (AAVs) are an integral part of gene therapy, yet engineering novel AAV variants is necessary to expand targetable tissues and treatable diseases. Directed evolution, particularly through DNA shuf...
Systemically delivered lipid nanoparticle-mRNA encoding lysosomal acid β-glucosidase restores the enzyme deficiency in a murine Gaucher disease model [0.03%]
系统递送的脂质纳米颗粒-mRNA编码酸性β-葡萄糖苷酶可恢复小鼠戈谢病模型中的酶缺陷
Yuanqing Liu,Shasha Wang,Yanni Chen et al.
Yuanqing Liu et al.
Gaucher disease (GD) is a rare genetically inherited illness caused by loss of lysosomal acid β-glucosidase (β-GCase) that leads to progressive accumulation of substrates, sphingolipid glucosylceramide (GL1) and glucosylsphingosine (lyso-...
Preclinical evaluation of lentiviral gene therapy for adenosine deaminase 2 deficiency (DADA2): engraftment efficiency and biodistribution in humanised NBSGW mice [0.03%]
adenosine deaminase 2缺乏症（DADA2）的慢病毒基因疗法的药代动力学和生物分布的临床前评估：人源化NBSGW小鼠中的植入效率和生物分布
Ying Hong,Alice Burleigh,Aiyin Liao et al.
Ying Hong et al.
Adenosine deaminase type 2 deficiency (DADA2) is caused by bi-allelic loss-of-function mutations in ADA2. While anti-TNF therapy is effective for the autoinflamatory and vasculitic components of the disease it does not correct marrow failur...
Mixture design as a tool for improving full-to-empty particle ratios across various GOIs in rAAV production [0.03%]
混合设计作为一种工具，可提高rAAV生产中各种GOI的全空颗粒比率
Konstantina Tzimou,Pol Hulsbus-Andreu,Ece Bahar Yildirim et al.
Konstantina Tzimou et al.
Optimization of recombinant adeno-associated virus (rAAV) production is essential for effective gene therapy applications. However, multiple factors affect the rAAV productivity in mammalian cells, and often they interact with each other, m...
Thrombotic microangiopathy following gene therapy for 5q-spinal muscular atrophy [0.03%]
基因治疗5q型脊髓性肌萎缩症后的微血管病变并发症
Clara Gontijo Camelo,Rodrigo Holanda Mendonça,Cristiane Araújo Martins Moreno et al.
Clara Gontijo Camelo et al.
Onasemnogene abeparvovec (OA) is the first gene replacement therapy (GT) approved for 5q spinal muscular atrophy (SMA). While effective, it can cause severe side effects, including thrombotic microangiopathy (TMA). The pathophysiology, risk...
Aptamer-targeted anti-miR RNA construct based on 3WJ as a new approach for the treatment of chronic kidney disease in an experimental model [0.03%]
基于三链体的适配体靶向抗微小核糖核酸构建体可作为实验性慢性肾脏病治疗的新方法
Aisha H A Alsenousy,Sara A Sharker,Mennatallah A Gowayed et al.
Aisha H A Alsenousy et al.
The treatment of chronic disease (CKD) is a great challenge in healthcare that requires an innovative approach to address its complex nature. RNA nanotechnology has emerged rapidly and received attention in the last few years because of its...
Engineered AAV capsids mediate transduction of murine neurofibroma and sciatic nerve [0.03%]
工程化AAV衣壳介导小鼠神经纤维瘤和腓肠神经转导
Edwina Abou Haidar,Shilpa Prabhakar,Pike See Cheah et al.
Edwina Abou Haidar et al.
Genetic diseases such as Neurofibromatosis type 1 (NF1) and Charcot-Marie Tooth disease involve Schwann cells (SCs) associated with peripheral nerves. Gene therapy using adeno-associated virus (AAV) vector mediated gene delivery is a promis...
RNAi targeting heparin cofactor II promotes hemostasis in a canine model of acquired hemophilia A [0.03%]
靶向肝素辅因子II的RNAi促进获得性A型血友病犬模型的止血
Yuyang Zhang,Tingting Liu,Haiming Kou et al.
Yuyang Zhang et al.
Heparin cofactor II (HCII) is a critical anticoagulant protein that inactivates thrombin. In our previous mouse studies, we demonstrated that GalNAc-HCII, a small interfering RNA (siRNA) targeting HCII conjugated with N-acetylgalactosamine ...
Recombinant oncolytic virus NDV-anti-VEGFR2 enhances radiotherapy sensitivity in NSCLC by targeting VEGF signaling and impairing DNA repair [0.03%]
重组抗血管生成肿瘤溶瘤病毒增强非小细胞肺癌放射治疗敏感性研究
Liang Liu,Liying Song,Tianyan Liu et al.
Liang Liu et al.
Resistance to radiotherapy is a significant challenge in the clinical management of non-small cell lung cancer (NSCLC). This study investigates a novel multimodal therapeutic strategy that combines oncolytic Newcastle disease virus (NDV) wi...
HDX-MS reveals pH and temperature-responsive regions on AAV capsids and the structural basis for DNA release [0.03%]
HDX-MS揭示AAV衣壳的pH和温度响应区域及其DNA释放的结构基础
Xiang Ye,Mengqi Hu,Yunli Hu et al.
Xiang Ye et al.
Recombinant adeno-associated viruses (AAVs) have become increasingly popular as gene therapy vectors in recent years. Like all viruses, AAVs undergo dynamic structural changes in response to varying temperature and pH conditions. However, t...