Results 71 to 80 of about 79,958 (292)

In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy

Molecular Therapy: Methods & Clinical Development, 2020
Delivery of therapeutic transgenes with adeno-associated viral (AAV) vectors for treatment of myopathies has yielded encouraging results in animal models and early clinical studies.
Jennifer B. Kwon, Adarsh R. Ettyreddy, Ashish Vankara, Joel D. Bohning, Garth Devlin, Stephen D. Hauschka, Aravind Asokan, Charles A. Gersbach   +7 more
doaj   +1 more source

A Toolkit for Targeted Neuromodulation of Striatal Direct Pathway Neurons Rescues Parkinsonian Motor Deficits in Mice

Advanced Science, EarlyView.
An adeno‐associated virus (AAV) toolkit enables selective anatomical and functional targeting of striatal D1‐MSNs through retrograde transduction. Enhanced capsids and engineered enhancers drive robust transgene expression across murine and primate models.
Zexuan Hong, Yujing Zhang, Junjiao Zhang, Hanhe Liu, Qiwei Liu, Yanglei Li, Lixin Yang, Lixia Li, Zhongjie Liu, Zhen Yuan, Zhonghua Lu, Yefei Chen, Yuantao Li, Yuwu Jiang, Taian Liu   +14 more
wiley   +1 more source

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries

Molecular Therapy: Methods & Clinical Development, 2020
Generation and screening of libraries of adeno-associated virus (AAV) variants have emerged as a powerful method for identifying novel capsids for gene therapy applications.
Pauline F. Schmit, Simon Pacouret, Eric Zinn, Elizabeth Telford, Fotini Nicolaou, Frédéric Broucque, Eva Andres-Mateos, Ru Xiao, Magalie Penaud-Budloo, Mohammed Bouzelha, Nicolas Jaulin, Oumeya Adjali, Eduard Ayuso, Luk H. Vandenberghe   +13 more
doaj   +1 more source

AAV and Insertional Mutagenesis

Molecular Therapy, 2007
In this issue of Molecular Therapy, David Russell provides an invited commentary on data presented at the tenth annual meeting of the American Society of Gene Therapy that strongly suggested that adenovirus-associated virus (AAV) vector insertion could in certain models be linked to the development of cancer.1 A short report describing the data that ...
openaire   +2 more sources

G3BP1 Succinylation at K413 is Critical for Cardiac Function by Modulating PI3K‐AKT‐mTOR Signal Axis

Advanced Science, EarlyView.
Schematic illustrating the impact of G3BP1 succinylation at K413 on cardiac function. In the healthy human heart, G3BP1 succinylation maintains homeostatic mTOR signaling. In patients with dilated cardiomyopathy (DCM) and heart failure (HF), G3BP1 de‐succinylation induces RagA expression and disrupts the binding of the TSC1/2 complex, leading to the ...
Yuan Zhang, Cancan Yao, Yan Chen, Ke Cai, Zhouping Lu, Boxuan Wu, Kun Yu, Yan Shi, Jianyuan Zhao, Xiangyu Zhou   +9 more
wiley   +1 more source

Nigra‐Subthalamic Dopaminergic Circuitry Modulates and Represents Distinct Pain Modality in Physiological and Pain States in Mice

Advanced Science, EarlyView.
Nigral dopaminergic (DA) neurons modulate and represent pain with a preference to a particular modality (mechanical) and laterality (contralateral), which are controlled by nigral GABAergic neurons. The pain modulation is mimicked by the nigro‐subthalamic projection and its downstream neurons, involving D2‐like receptors.
Ying Ji, Shuyi Li, Jiaqi Zhang, Xiang‐Ying Xu, Cui Yin, Mu Niu, Danyang Liu, Yanyu Jiang, Yong Niu, Su Liu, Bin Zhang, Guiyun Cui, Chunyi Zhou, Cheng Xiao   +13 more
wiley   +1 more source

FGF13 Deficiency Ameliorates Paclitaxel‐Induced Neuropathic Pain by Inhibiting VASH1‐Mediated Microtubule Detyrosination to Promote Mitophagy

Advanced Science, EarlyView.
FGF13 is upregulated in DRG neurons of PIPNP model mice. DRG neuron‐specific knockout of FGF13 ameliorates PIPNP symptoms. Mechanistically, FGF13 potentiates microtubule detyrosination by promoting VASH1 binding to microtubules. FGF13 knockout suppresses VASH1‐mediated microtubule detyrosination and promotes α‐tubulin tyrosination.
Yiming Dong, Yidan Wang, Simeng Lv, Zishan Dong, Kaixi Zhi, Xiuhua Guo, Xuyan Li, Ruoxi Yu, Yiyi Zhang, Siyuan Cheng, Chuan Wang   +10 more
wiley   +1 more source

An Activity‐Dependent NEPAS–PTX3 Axis Links Neurovascular and Myelin Deficits to Cognitive Impairment

Advanced Science, EarlyView.
An activity‐dependent pathway links prefrontal circuit hypoactivity to cognitive impairment. Reduced PVA–mPFC activity upregulates NEPAS, which suppresses PTX3 secretion, leading to impaired angiogenesis, myelin deficits, and memory decline. Rescue is achieved by NEPAS knockdown or chemogenetic circuit activation.
Boya Hu, Zifei Chen, Bingmei Sun, Jiale Gao, Jiale Xu, Xiaochun Guo, Fenfei Gao, Zhongsi Wang, Jie Wu, Xiaoyu Ji, Peipei Liu, Bing Huang   +11 more
wiley   +1 more source

A Generic Method for Fast and Sensitive Detection of Adeno-Associated Viruses Using Modified AAV Receptor Recombinant Proteins

Molecules, 2019
Adeno-Associated Viruses (AAV) are widely used gene-therapy vectors for both clinical applications and laboratory investigations. The titering of different AAV preparations is important for quality control purposes, as well as in comparative studies ...
Mengtian Cui, Yabin Lu, Can Tang, Ran Zhang, Jing Wang, Yang Si, Shan Cheng, Wei Ding   +7 more
doaj   +1 more source

Targeting Supramolecular Active Complexes of Nav1.7/Nav1.8 to Relieve Chronic Neuropathic Pain

Advanced Science, EarlyView.
In mice and patients with severe chronic neuropathic pain (NP), Nav1.7, Nav1.8, TrkB, and five cytoskeletal proteins form supramolecular active complexes (SMACs) with polygonal lattice structures as noxious signal amplifiers in dorsal root ganglion (DRG) neurons.
Liting Sun, Hang Xian, Yunxin Shi, Taotan Yang, Hongyan Shuai, Wenchao Hu, Siying Fei, Miao Xu, Taoyuan Yang, Ruilong Xia, Ting Wen, Fengting Zhu, Yan Fu, Yang Li, Wei Xia, Ran Qian, Yuanying Liu, Zhicheng Tian, Lamei Li, Qian Zhou, Lize Xiong, Rui Cong, Ceng Luo, Shengxi Wu, Xiafeng Shen, Xin Yu, Rou‐Gang Xie, Changgeng Peng   +27 more
wiley   +1 more source