Results 81 to 90 of about 326,770 (247)

Vaccinia virus as a subhelper for AAV replication and packaging

Molecular Therapy: Methods & Clinical Development, 2015
Adeno-associated virus (AAV) has been widely used as a gene therapy vector to treat a variety of disorders. While these vectors are increasingly popular and successful in the clinic, there is still much to learn about the viruses.
Andrea R Moore, Biao Dong, Lingxia Chen, Weidong Xiao   +3 more
doaj   +1 more source

USP30‐Mediated Deubiquitination of PEX5 Suppresses Pexophagy to Drive Tubular Injury in Diabetic Kidney Disease

Advanced Science, EarlyView.
USP30 accumulation in diabetic renal tubular epithelial cells deubiquitinates PEX5, suppresses pexophagy, and drives dysfunctional peroxisome accumulation and tubular injury. USP30 depletion restores PEX5 ubiquitination and pexophagy, improves peroxisomal function, and alleviates tubular damage, highlighting USP30 as a therapeutic target in diabetic ...
Jia Li, Chaoyang Hua, Guangpu Li, Furong Zhai, Jingjing Ren, Shaokang Pan, Qi Feng, Jianguo Wen, Dongwei Liu, Zhangsuo Liu, Jiayu Duan   +10 more
wiley   +1 more source

Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells [PDF]

, 2019
Adeno-associated virus (AAV) vectors are currently among the most commonly applied for in vivo gene therapy approaches. The evaluation of vectors during clinical development requires the production of considerable amounts of highly pure and potent ...
Rey, Maria, Deglon, Nicole, Blessing, Daniel, Vachey, Gabriel, Pythoud, Catherine, Wurm, Florian M., Padrun, Vivianne, Schneider, Bernard L., Rey M, Padrun, V., Blessing D, Schneider BL, Pythoud C, Déglon, N., Rey, M., Blessing, D., Vachey G, Vachey, G., Padrun V, Déglon N, Wurm FM, Wurm, F.M., Schneider, B.L., Pythoud, C.   +23 more
core   +1 more source

TAB2 Causes Neuronal Damage by Aggravating Microglia‐Mediated Neuroinflammation in Parkinson's Disease

Advanced Science, EarlyView.
In microglia, STAT3 upregulates TAB2, which promotes NF‐κB activation through its NZF domain‐mediated recognition of K63‐linked ubiquitin chains, leading to inflammatory cytokine release and subsequent neuronal injury. Lumacaftor suppresses TAB2 expression and directly binds the TAB2‐NZF domain to interrupt K63 ubiquitin recognition, thereby blocking ...
Yanhao Zhao, Zimeng Huang, Jianing Gao, Aolan Li, Huizhi Wang, Yingchuan Chen, Guanyu Zhu, Xin Zhang, Yin Jiang, Moxuan Zhang, Fangang Meng, Jianguo Zhang, Tingting Du   +12 more
wiley   +1 more source

Loss of LCN2 Function Ameliorates Glucocorticoid‐Induced Muscle Atrophy via Remodeling the Extracellular Matrix

Advanced Science, EarlyView.
Glucocorticoids transcriptionally activate LCN2 expression via GR nuclear translocation. Secreted LCN2 binds MMP9 to degrade skeletal muscle ECM collagen, blocks integrin‐mediated mechanotransduction, bidirectionally disrupts muscle protein homeostasis, and reveals a novel target for steroid‐induced muscle atrophy.
Hongwei Shi, Xiaojing Hao, Yi Yan, Fengyang Li, Yaru Zhang, Zixin Li, Wenjuan Xie, Jiayin Lu, Xiaomao Luo, Yanjun Dong, Haidong Wang, Juan Wang   +11 more
wiley   +1 more source

Fzd7 Restrains Pink1‐Dependent Mitophagy in Suture Stem Cells to Maintain Cranial Suture Patency

Advanced Science, EarlyView.
How suture stem cells fail to preserve cranial suture patency remains incompletely understood. Integrated single‐cell and high‐resolution spatial transcriptomic analyses identify reduced Fzd7 expression in Prrx1+ suture stem cells as an early feature of craniosynostosis.
Xinyan Chen, Zong Chen, Chenzhi Lai, Yingying Yue, Tian He, Hao Gong, Jing Shi, Xiaoshuang Guo, Yu Wang, Guodong Song, Xiaolei Jin   +10 more
wiley   +1 more source

Adeno‐associated virus vectors for gene therapy—focusing on melanoma

Interdisciplinary Medicine
Adeno‐Associated Virus (AAV) vectors have been found to have great potential in the field of gene therapy due to their unique properties. These nonpathogenic vectors exhibit high tissue specificity, low immunogenicity, and sustained gene expression ...
Xingyue Wang, Miao Wei, Rui Miao, Xiujing Hao, Min Li, Wenxin Wang, Zhonglei He   +6 more
doaj   +1 more source

Development and application of non-integrating lentiviral vectors for gene therapy [PDF]

, 2009
Lentiviruses stably integrate their genome into the host genome. Although this feature can be advantageous for long term transgene expression, it also has the potential to cause mutagenesis and cell transformation.
Apolonia, L.F.S.
core  

Endothelial-to-mesenchymal transition enhances permissiveness to AAV vectors in cardiac endothelial cells [PDF]

A major obstacle in inducing therapeutic angiogenesis in the heart is inefficient gene transfer to endothelial cells (ECs). Here, we identify compounds able to enhance the permissiveness of cardiac ECs to adeno-associated virus (AAV) vectors, which stand
Vuerich, Roman, Marengon, Margherita, Volpe, Maria Concetta, Zentilin, Lorena, Ring, Nadja Anneliese Ruth, Vodret, Simone, Zacchigna, Serena, Volf, Nina, Colliva, Andrea, Giacca, Mauro, Braga, Luca   +10 more
core   +1 more source

Phase Separation of TRIM21 Modulates PTPN14 Stability to Drive Flow‐Dependent Endothelial Activation and Atherogenesis

Advanced Science, EarlyView.
Disturbed flow promotes the formation of TRIM21‐rich biomolecular droplets, which concentrate TRIM21 and PTPN14 and facilitate their SPRY‐FERM interaction (illustrated by the TRIM21 D355‐PTPN14 R132 salt bridge). This condensate‐driven proximity enables TRIM21 to catalyze K48‐linked polyubiquitination of PTPN14 at lysine 956, leading to proteasome ...
Xue He, Qiannan Ma, Xue Wang, Jiaxu Mang, Chufeng Li, Bingjun Wu, Liu Yao, Yequn Chen, Qiankun Bao, Yi Zhu, Jinlong He   +10 more
wiley   +1 more source