Advanced iPSC-based modeling of LMNA-related congenital muscular dystrophy enables development of genetic therapies for muscle laminopathies [0.03%]
Daniel Moore,Heather Steele-Stallard,Luca Pinton et al.
Daniel Moore et al.
LMNA-related congenital muscular dystrophy (L-CMD) is one of the most severe laminopathies, which are incurable diseases primarily caused by pathogenic LMNA variants. LMNA encodes Lamin A/C, key components of the nuclear lamina, which provi...
Intranasal delivery of a vasoactive intestinal peptide-based circRNA vaccine induces systemic and mucosal immunity against RSV in mice [0.03%]
Junqi Xiang,Jie Xu,Jing Zhu et al.
Junqi Xiang et al.
Respiratory syncytial virus (RSV) is a major cause of acute lower respiratory tract disease in infants and older adults; however, vaccine strategies capable of inducing robust respiratory mucosal immunity remain limited. Here, we developed ...
Yuanyuan Wang,Qingtao Sun,Wenqiang Zheng et al.
Yuanyuan Wang et al.
Mounting evidence indicates that interleukin-6 (IL-6) plays an essential role in the development of cancer cachexia. Particularly, recent work showed that IL-6 drives cancer cachexia through neurons in the area postrema of the brainstem. Ho...
Leveraging host-cell modulators of adeno-associated vector transduction to tailor viral biodistribution [0.03%]
Katie Kubek-Luck,Jason Velazquez,Xiaorui Yao et al.
Katie Kubek-Luck et al.
Clinical successes in gene therapy using adeno-associated vectors (AAVs) are offering a hopeful path toward the correction of several monogenic disorders. At its core, AAV gene delivery relies on multiple interactions between the capsid or ...
Shufeng Feng,Lu Cui,Yongxin Zhu et al.
Shufeng Feng et al.
Oxidative stress, a pervasive cancer vulnerability, remains a challenging therapeutic target attributed to tumor heterogeneity and adaptive resistance. Herein, we identify RhoA as a "redox rheostat" during oncolytic virotherapy by regulatin...
DC vaccine loaded with Bacteroides fragilis elicits functional cross-reactivity and enhances anti-PD-1 immunotherapy [0.03%]
脆弱拟杆菌载荷的瘤内注射细菌DNA疫苗诱导功能交叉反应并增强抗PD-1免疫治疗
Shichuan Hu,Gang Shi,Jing Zhao et al.
Shichuan Hu et al.
The gut microbiome profoundly influences the clinical benefits of antitumor immunotherapy. Although various gut microbiota have been shown to enhance immunotherapy, their clinical application remains unexplored. In this study, a dendritic c...
DC vaccine loaded with Bacteroides fragilis elicits functional cross-reactivity and enhances anti-PD-1 immunotherapy [0.03%]
脆弱拟杆菌载荷的肿瘤疫苗诱导功能交叉反应并增强抗PD-1免疫疗法
Shichuan Hu,Gang Shi,Jing Zhao et al.
Shichuan Hu et al.
The gut microbiome profoundly influences the clinical benefits of antitumor immunotherapy. Although various gut microbiota have been shown to enhance immunotherapy, their clinical application remains unexplored. In this study, a dendritic c...
Viral gene replication enhances AAV vector quality and reduces manufacturing costs [0.03%]
病毒基因复制可提高AAV载体质量并降低制造成本
Hao Liu,Ailing Du,Nan Liu et al.
Hao Liu et al.
Viral vectors for gene and cell therapy are typically produced by transfecting plasmids into mammalian cells. Because plasmid DNA is not replicated in these cells, large quantities are required to express sufficient viral proteins for vecto...
Viral gene replication enhances AAV vector quality and reduces manufacturing costs [0.03%]
病毒基因复制可提高AAV载体质量并降低制造成本
Hao Liu,Ailing Du,Nan Liu et al.
Hao Liu et al.
Viral vectors for gene and cell therapy are typically produced by transfecting plasmids into mammalian cells. Because plasmid DNA is not replicated in these cells, large quantities are required to express sufficient viral proteins for vecto...
Engineering T cells in vivo to express a synthetic cytokine receptor enables preferential expansion and enrichment of anti-CD22 CAR T cells [0.03%]
体内工程改造T细胞表达合成细胞因子受体，以实现抗CD22嵌合抗原受体T细胞的优先扩增和富集
Travis J Friesen,Alyssa Sheih,Nikole Perdue et al.
Travis J Friesen et al.
Ex vivo-manufactured chimeric antigen receptor (CAR) T cells targeting CD19 have transformed treatment strategies for patients with B cell malignancies and shown promising results in autoimmune disease. Yet despite their success, significan...