Results 121 to 130 of about 326,770 (247)

AAV6 vectors provide superior gene transfer compared to AAV9 vectors following intramyocardial administration

Molecular Therapy: Methods & Clinical Development
Cardiac gene therapy using adeno-associated viral (AAV) vectors holds great promise for treating heart diseases but would benefit from more potent AAV vectors. Vectors based on the AAV serotypes 6 and 9 have been used in pre-clinical gene therapy studies,
Jianan Wang, Timo Jonker, Aina Cervera-Barea, Zhenyu Dong, Ruud N. Visser, Evelien E. Birza, Arie R. Boender, Mischa Klerk, Yuting Yang, Joyce Visser, Marlijn S. Jansen, Tom C. Grootswagers, Cindy I. Bart, Saskia C.A. de Jager, Silke Schrödel, Christian Thirion, Osne F. Kirzner, Hanno L. Tan, Antoine A.F. de Vries, Joost P.G. Sluijter, Klaus Neef, Joris R. de Groot, Vincent M. Christoffels, Gerard J.J. Boink   +23 more
doaj   +1 more source

AAV Vectors, Insertional Mutagenesis, and Cancer

Molecular Therapy, 2007
By now we have all heard a lot about retroviral vectors and cancer, but other types of vectors have typically been left out of these discussions. However, results presented at the recent American Society of Gene Therapy meeting and just published in Science1 show that this is a subject that goes beyond retroviruses, hematopoietic cells, and gamma C ...
openaire   +2 more sources

Development of Adeno-associated viral (AAV) vectors that specifically express in endothelial cells [PDF]

, 2019
Optimization of transduction of Adeno-associated viral (AAV) vectors that specifically express in endothelial cells with an element of a super ...
Esparta González, Olaya
core  

AAV-DN-TNF-GFP and AAV-GFP vector injections resulted in sustained transgene expression in the striatum.

, 2014
(A) GFP expression in a 12-month animal that received AAV-GFP vector injection. (B) Higher magnification view of boxed area. AAV vectors transduced (C) neurons and (D) glia (arrows). (E) DN-TNF ELISA using dissected striata from 3, 9 and 12-month animals
Kelly A. Ruhn (83723), Malú G. Tansey (83727), Xi Chen (35903), Laura Taylor Alto (563220), Isaac Treviño (83726)   +4 more
core   +1 more source

The GPX3‐VCAM1 Axis Gates Pro‐Fibrotic Tubule Cell Fate in Hyperuricemic Nephropathy

Advanced Science, EarlyView.
Hyperuricemia induces NLRP3 inflammasome signaling activation and downregulation of GPX3 in proximal tubular epithelial cells, leading to oxidative stress–driven VCAM1 upregulation, emergence of pro‐fibrotic proximal tubule cells, macrophage recruitment, and renal fibrosis.
Yunfei Qi, Qiang Zhao, Yue Lve, Yaming Yuan, Tingting Zhou, Yue Yuan, Dexi Wu, Yifan Tang, Yan Liu, Zhigang Li, Chunping Jiang, Qiurong Ding   +11 more
wiley   +1 more source

CRISPR and Gene Augmentation Rescue Trabecular Meshwork Dysfunction in iPSC Models of Lowe Syndrome

Advanced Science, EarlyView.
By modeling Lowe syndrome using patient‐derived iPSCs, this study establishes a human disease model that faithfully recapitulates OCRL deficiency‐associated ciliary and cytoskeletal defects. The model enables evaluation of both mutation‐agnostic DNA augmentation and CRISPR‐mediated mutation correction strategies, both of which restore OCRL function and
Siyu Chen, Zhiquan Liu, Wenmin Wang, Qing Wang, Tia J. Kowal, Fan Zhang, Grzegorz Walkiewicz, Faruk Hossen, Herbert M. Lachman, Jinqiong Zhou, Yiting Wang, Yang Sun   +11 more
wiley   +1 more source

Palmitoyl Acyltransferase Zdhhc17 Promotes Functional Recovery After Spinal Cord Injury by Targeting the Nuclear Transport Factors Kpna2 and Ipo9

Advanced Science, EarlyView.
In this study, we demonstrate that Zdhhc17, acting as a PAT, suppresses neuronal apoptosis and promotes axon regeneration after injury, thereby enhancing functional recovery following SCI. In this context, Kpna2 and Ipo9 serve as novel palmitoylation substrates of Zdhhc17, whose palmitoylation prevents the injury‐induced degradation of their proteins ...
Meixuan Chen, Huan Chen, Sining Ding, Fan Liu, Xiaofei Zheng, Yuan Wang, Ruitu Tian, Huan Li, Peilin Liu, Le Hu, Bin Liu, Limin Rong, Mangmang Li   +12 more
wiley   +1 more source

Neuron‐Derived Vesicular circMagi1 Restrains Vascular Repair After Ischemic Stroke by Promoting TRIM65‐Mediated ANXA2 Degradation

Advanced Science, EarlyView.
After ischemic stroke, injured neurons transfer circMagi1 to endothelial cells through extracellular vesicles. Within endothelial cells, circMagi1 binds ANXA2 and enhances its engagement with TRIM65, promoting ANXA2 ubiquitination and proteasomal degradation to constrain angiogenic repair.
Youkun Bi, Qun Liu, Baohua Zou, Wenyuan Wang, Xinlong Qiao, Lin Gao, Chaoxu Miao, Ningning Huang, Shuqing Tang, Rui Guo, Changqing Zeng, Ce Jia, Yuanchao Xue, Xuejun Guo, Jianjun Chen, Huiwen Wang, Guangju Ji   +16 more
wiley   +1 more source

Hederagenin Attenuates Morphine Addiction via GABAA1‐Mediated Modulation of Ca2+–CaMK–CREB Signaling in Ventral Tegmental Dopaminergic Neurons

Advanced Science, EarlyView.
Hederagenin is identified as a GABAA1‐targeting natural compound that suppresses morphine‐associated reward by restraining Ca2+–CaMK–cAMP–CREB signaling in ventral tegmental dopaminergic neurons. RVG29‐modified liposomal delivery further enhances brain accumulation and behavioral efficacy, providing a mechanistically defined strategy for natural ...
Hongyang Jiang, Ziting Zhou, Nannan Xue, Jiasi Deng, Yuanyuan Zhou, Xinying Lv, Yuxuan Wang, Yi Zhang, Haotian Pan, Geyan Xu, Yinkun Tao, Mengran Li, Xin Cui, Hongyue Ma, Qian Wang, Zhigang Lu   +15 more
wiley   +1 more source

A Gut‐Brain Axis Underlying Succinate‐Associated Compulsive‐Like Behavior

Advanced Science, EarlyView.
Gut‐derived succinate is associated with an excitatory‐inhibitory imbalance in dmPFC glutamatergic neurons, involving microglial SUCNR1 activation and vagal afferent signaling via upregulated gut 5‐HT4 receptors along the DVC‐CeA‐dmPFC circuit, changes that are linked to excessive compulsive‐like behavior.
Yi Li, Ying‐Dan Zhang, Wei Wang, Xin‐Yi Wu, Di Li, Xin Gong, Fei Tian, Meng‐Yu Dai, Jian Gao, Zi‐Feng Zheng, Zi‐Yang Bi, Dong‐Dong Shi, Zhen Wang   +12 more
wiley   +1 more source