Results 91 to 100 of about 326,770 (247)

mGem: AAV, from almost a virus to an awesome vector—or is it?

mBio
Adeno-associated virus (AAV) vectors have taken center stage for gene therapy and have shown clinical efficacy in 15 human diseases to date. The Food and Drug Administration has approved seven AAV “drugs” for one-time treatment respectively for Leber’s ...
Arun Srivastava
doaj   +1 more source

Tumor-Specific Delivery of Immune Checkpoint Inhibitors by Engineered AAV Vectors [PDF]

, 2019
Immune checkpoint inhibitors (ICIs) can block distinct receptors on T cells or tumor cells thus preventing T cell inactivation and tumor immune escape. While the clinical response to treatment with ICIs in cancer patients is impressive, this therapy is ...
Christine E. Engeland, Johanna Reul, Guy Ungerechts, Jessica Hartmann, Janina Frisch, Christian J. Buchholz, Frederic B. Thalheimer   +6 more
core   +1 more source

Compact 9dBEs Enable Efficient and Precise Genome Editing in Mammalian Cells and In Vivo

Advanced Science, EarlyView.
As a compact type II‐D system, the Cas9d‐based platform holds great potential for in vivo applications. Through rational engineering, its derived base editors (9dBEs) enable efficient disease modeling while facilitating single‐vector AAV delivery for in vivo genome editing. These miniature tools offer a robust strategy for basic research and biomedical
Qingquan Xiao, Zhijin Tian, Luqi Weng, Kun Xu, Dingyi Han, Zhaowei Wu, Junnan Li, Shangpu Li, Qijing Zhang, Xiaohui Zhao, Guoling Li, Yinghui Wei, Zhen Gu   +12 more
wiley   +1 more source

Gut Microbiota‐Isoallolithocholic Acid Crosstalk Promotes Calcium Oxalate Kidney Stone Formation via PARP1‐Mediated Parthanatos

Advanced Science, EarlyView.
ABSTRACT Bile acids have been implicated in calcium oxalate (CaOx) nephrolithiasis. Here, we employ multi‐omics approaches to identify isoallolithocholic acid (isoalloLCA) as the key bile acid elevated in the feces, serum, kidney, and urine of CaOx rats, confirmed by spatial metabolomics.
Zijian Zhou, Ruixi Yu, Xuan Zhou, Feng Shi, Zeyu Lin, Peng Gao, Lujia Wang, Ninghan Feng, Zhong Wu, Dawei Wang   +9 more
wiley   +1 more source

Macrophage PABPC4‐SPP1 Axis Orchestrates Immunosuppression in Colorectal Cancer

Advanced Science, EarlyView.
In the CRC microenvironment, macrophage PABPC4 binds to the 3′UTR of SPP1 mRNA to stabilize its expression, thereby sustaining M2‐like immunosuppressive macrophage polarization. Concurrently, this axis suppresses CD8+ T cell effector functions via CD44 signaling, collectively fostering an immunosuppressive niche that drives tumor progression.
Meng Wang, Fang Yang, Yuan Gao, Liuli Li, Yulan Huang, Hui Yao, Sijie Zhao, Kun Zhao, Tianying Zhang, Hui Heng, Jiaxin Xu, Yuancai Xiang, Weidong Xiao, Shaofan Hu, Hongming Miao   +14 more
wiley   +1 more source

KLF5 Downregulation Links Impaired BNIP3‐Mediated Mitophagy to Inflammatory Valve Remodeling in Calcific Aortic Valve Disease

Advanced Science, EarlyView.
In human CAVD, KLF5 is reduced in VIC‐rich regions and remodeling/stress‐associated VIC states. In VICs, KLF5 sustains BNIP3 promoter activity and BNIP3‐mediated mitophagy, thereby limiting cytosolic mtDNA accumulation. KLF5 loss weakens mitochondrial quality control and enhances mtDNA‐sensitive STING/NF‐κB/NLRP3 inflammatory signaling under osteogenic
Jin‐Hui Bian, Jia‐Xi Gu, Zhang Yue, Chun‐Ze Yuan, Hai‐Bin Ji, Ao Li, Yu Zhong, Xiu‐Fan Xu, Yu‐Qiu He, Ye‐Fan Jiang, Hong‐Shan Chen, Xiang Chen, Meng‐Lin Chao, Yong‐Feng Shao   +13 more
wiley   +1 more source

SerpinA3 is an Endogenous TGF‐β Receptor Antagonist that Attenuates Cardiac Fibroblast Activation and Fibrotic Remodeling

Advanced Science, EarlyView.
SerpinA3 acts as an endogenous TGF‐β receptor antagonist that binds to the extracellular domain of TGFR‐1, thereby preventing TGFR‐1–TGFR‐2 complex formation. This receptor‐level blockade suppresses TGF‐β/Smad2/3 signaling, attenuates cardiac fibroblast activation, and extracellular matrix deposition, and ultimately alleviates pressure overload–induced
Hui Wang, Jieyu Cui, Chunlu Huang, Wanyi Guo, Tong Liu, Yi Zhu, Shuai Shao, Kangyin Chen, Jinlong He, Qiankun Bao   +9 more
wiley   +1 more source

Next-generation strategies to improve safety and efficacy of adeno-associated virus-based gene therapy for hemophilia: lessons from clinical trials in other gene therapies

Haematologica
Three major directions for the global progress of adeno-associated virus (AAV) vectors for gene therapies (GT) are analyzed: a) engineering vectors to increase transgene expression; b) aligning interests of the health system with costs and challenges ...
Giovanni Di Minno, Wolfgang Miesbach, Giancarlo Castaman, Flora Peyvandi   +3 more
doaj   +1 more source

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Nature Communications, 2018
Immunogenicity of AAV vectors renders repeated AAV dosing ineffective. Here the authors show that coadministration of nanoparticle-encapsulated rapamycin overcomes AAV immunogenicity through Treg induction, enabling efficient AAV redosing in mice and ...
Amine Meliani, Florence Boisgerault, Romain Hardet, Solenne Marmier, Fanny Collaud, Giuseppe Ronzitti, Christian Leborgne, Helena Costa Verdera, Marcelo Simon Sola, Severine Charles, Alban Vignaud, Laetitia van Wittenberghe, Giorgia Manni, Olivier Christophe, Francesca Fallarino, Christopher Roy, Alicia Michaud, Petr Ilyinskii, Takashi Kei Kishimoto, Federico Mingozzi   +19 more
doaj   +1 more source

An RNA‐Binding Protein RBMS1 Promotes Endothelial Cell Autophagy to Maintain Vascular Homeostasis and Suppress Deep Vein Thrombosis

Advanced Science, EarlyView.
RBMS1, an RNA‐binding protein, restores vascular endothelial homeostasis by enhancing ATG3‐mediated autophagy, thereby attenuating thrombus formation and suggesting a potential avenue for further therapeutic exploration in DVT. ABSTRACT Deep vein thrombosis (DVT) is a prevalent vascular disorder characterized by aberrant coagulation within the deep ...
Chu Chu, Xinkui Liu, Nannan Fan, Xiaoyan Yu, Qiaoqiao Han, Yunhong Zhang, Wei Li, Yongqing Cai, Miaomiao Zhou, Fang Li, Feifei Shi, Zhen Zhang, Bin Wang, Xia Li   +13 more
wiley   +1 more source