Results 61 to 70 of about 32,863 (259)

Reducing the Risk of Adeno-Associated Virus (AAV) Vector Mobilization with AAV Type 5 Vectors [PDF]

Journal of Virology, 2009
ABSTRACT Current adeno-associated virus (AAV) gene therapy vectors package a transgene flanked by the terminal repeats (TRs) of AAV type 2 (AAV2). Although these vectors are replication deficient, wild-type (wt) AAV2 prevalent in the human population could lead to replication and packaging of a type 2 TR (TR2)-flanked transgene ...
F Curtis, Hewitt, Chengwen, Li, Steven J, Gray, Shelley, Cockrell, Michael, Washburn, R Jude, Samulski   +5 more
openaire   +2 more sources

TOLLIP Inhibits Psoriasis Progression via Suppressing PKM2‐Mediated Glycolysis in Keratinocytes

Advanced Science, EarlyView.
In this study, we identify TOLLIP as a critical regulator of psoriasis pathogenesis through its modulation of glycolytic metabolism. Our findings establish the TOLLIP‐PKM2‐glycolysis axis as a key mechanism linking metabolic reprogramming to psoriasis pathogenesis, and propose TOLLIP as a promising therapeutic target.
Xiuhuan Jiang, Guoying Miao, Na Shen, Qingxia Han, Xiaoru Han, Zichang Qiao, Kexin Feng, Zhuo Tan, Yaguang Wang, Xiuhua Liu, Chen Wu, Zhenzhen Yan   +11 more
wiley   +1 more source

BIN1 and ALDH1B1 Deficiency in Colonic Smooth Muscle Drives Mitochondrial Dysfunction and Fibrosis in Slow‐Transit Constipation

Advanced Science, EarlyView.
Slow‐transit constipation (STC) is a disabling motility disorder with unclear smooth‐muscle mechanisms. Spatial proteomic analysis of STC patient colon reveals both the central pathogenic role of smooth muscle cells (SMCs) in STC and novel regulators of intestinal motility, BIN1 and ALDH1B1.
Jianbo Liu, Hao Zhang, Wenhao Qiao, Ran Liu, Jie Sun, Xiaopei Li, Qin Li, Dongbo Zhao, Dawei Chen, Jingxin Li, Shuxiao Dong   +10 more
wiley   +1 more source

Advances in Bioanalytical Techniques and Pharmacokinetics/Pharmacodynamics Research of Adeno-Associated Viral Vector Gene Therapy Drugs

罕见病研究
Recently, adeno-associated virus(AAV)vectors have been a hotspot in gene therapy and have shown great potential for rare diseases therapy, due to their low pathogenicity, mild immunogenicity and high tissue specificity.
REN Jianwei, ZHENG Xin, HAN Xiaohong
doaj   +1 more source

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

Palatable‐Food–Driven Top‐Down Circuit Inhibits PVNCRF Activity to Mitigate Stress Via Peri‐PVNCRFR1 Neurons

Advanced Science, EarlyView.
Palatable food alleviates stress and prevents anxiety. This study uncovers a dedicated neural pathway: dopamine release in the PFC activates D1R neurons, whose projections to the peri‐PVN engage a population of anxiolytic CRFR1 neurons. These neurons then inhibit stress‐induced hyperactivity of PVNCRF neurons, providing a circuit‐level explanation for ...
Yuchuan Hong, Shirui Jun, Tianjiao Deng, Gaojie Shao, Dan Liu, Yi Sun, Yan Chen, Qian Xiao, Jie Shao, Sheng Wang, Tianwen Huang, Fan Yang, Jie Tu   +12 more
wiley   +1 more source

Herba Lysimachiae Polysaccharide‐Modified Selenium Nanoparticles Alleviate Oxidative Injury in Kidney Stones via TOMM22‐Regulated Mitophagy Activation

Advanced Science, EarlyView.
HLP‐SeNPs, a Lysimachia christinae polysaccharide‐modified selenium nanoparticle, treat kidney stones by downregulating TOMM22 expression, which reduces PINK1 translocation into mitochondria and promotes its retention on the outer mitochondrial membrane.
Junyi Yang, Guoruiyu Lyu, Wenlong Wan, Dongfeng Yuan, Jiabo Li, Yongqi Wang, Baokang Wang, Zhilong Ma, Yuanyuan Yang, Yang Xun, Xiao Yu   +10 more
wiley   +1 more source

NAT10‐Mediated ac4C Modification of circANKRD12 Reprograms the Tumor Microenvironment

Advanced Science, EarlyView.
NAT10‐dependent acetylation of circANKRD12 drives translation of the circANKRD12_354aa protein, which binds HDAC2 to stabilize c‐Myc via deubiquitination, promoting multiple myeloma (MM) cell proliferation. Concurrently, the circANKRD12‐HDAC2 axis suppresses H3ac‐mediated transcription of IFN‐γ, TNF‐α, and GZMB in NK cells, leading to NK cell ...
Jiale Zhang, Hui Shi, Chen Wang, Zihao Liu, Lianxin Zhou, Xinyu Lv, Mengjie Guo, Ye Yang, Chunyan Gu   +8 more
wiley   +1 more source

Renal IGFBP6 Interacts With THBS1 to Drive Renal Cellular Senescence and Fibrosis

Advanced Science, EarlyView.
ABSTRACT Epithelial dedifferentiation and myofibroblast activation are critical drivers of chronic kidney disease (CKD) progression. Elevated levels of IGFBP6 have been linked to decreased renal function in CKD patients, but its precise role and underlying mechanisms remain unclear.
Ju‐tao Yu, Chang‐geng Xu, Xiao‐wei Hu, Run‐run Shan, Jia‐nan Wang, Xiang‐yu Li, Yu‐qing Wang, Yu‐hang Dong, Shen‐Zhuo‐Fan Huang, Shuai Sun, Tian Zhao, Ya‐Xin Ru, Xiao‐yu Shen, Rui Hou, Jian‐min You, Fei Zhang, Chao Li, Jie Wei, Chao Hou, Zhi‐juan Wang, Dan‐feng Zhang, Juan Jin, Jia‐gen Wen, Yu‐jie Liu, Ming‐ming Liu, Xiao‐ming Meng, Wei Wang   +26 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