Results 71 to 80 of about 9,649,206 (293)

Mutation-independent treatment of autosomal dominant Retinitis Pigmentosa (adRP) [PDF]

, 2010
Viral-mediated gene therapy holds great promise for the treatment of severe inherited retinal diseases, such as Retintitis Pigmentosa (RP), which is caused by mutations in genes preferentially expressed in photoreceptor cells. The availability of vectors
Mussolino, Claudio
core   +1 more source

A Rare De Novo Missense Mutation in IFT122 Confers a Genetic Susceptibility Factor of Idiopathic Pediatric Uveitis Via Trio‐based Whole‐Exome Sequencing

Advanced Science, EarlyView.
A rare de novo IFT122‐A773E variant is identified in idiopathic pediatric uveitis and shown to exacerbate retinal inflammation and barrier dysfunction. Mechanistically, the variant enhances IFT43 interaction, elevates calcium signaling, and activates the MEK/ERK/FRA1 axis, revealing a previously unrecognized cilia‐associated pathway that may increase ...
Qian Zhou, Jiaxing Huang, Yilin Wang, Xingran Li, Xianyang Liu, Lingyu Dai, Hongshun Li, Qingfeng Wang, Jiangyi Liu, Guannan Su, Wanyun Zhang, Yang Deng, Qingfeng Cao, Yujie Lai, Xiang Luo, Changwei Huang, Ling Chen, Shengping Hou, Peizeng Yang   +18 more
wiley   +1 more source

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]

, 2002
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Hanawa, H, Ng, CYC, Mandrell, TD, Vanin, EF, Nathwani, AC, Hu, Y, Davidoff, AM, Hoffer, FA, Zhou, J, Slaughter, C, Nikanorov, A, Lozier, JN, Nienhuis, AW   +12 more
core  

Entorhinal Astrocyte Transplants Restore Spatial Exploration and Alleviate Amyloid‐Beta Pathology in Alzheimer's Mice

Advanced Science, EarlyView.
Impaired MEC astrocytic Ca2+ signaling is associated with fragmented spatial exploration in AD mice. Region‐specific glial progenitor transplantation generates engrafted astrocytes that are accompanied by improved AQP4 polarization, reduced amyloid‐β‐associated pathology, attenuated neuroinflammation, preserved synaptic integrity, and ameliorated ...
Fengjuan Wu, Jihua Fan, Xiang Zhou, Zhiqi Yang, Mingyue Gong, Mingzhu Huang, Zhenlu Cai, Shaofan Yang, Teng Teng, Chuanyan Yang, Jin Li, Haoyu Wang, Shuangshuang Dai, Chunhai Chen, Xiaowei Chen, Manxia Wang, Kuan Zhang   +16 more
wiley   +1 more source

Identification of relevant analytical methods for adeno-associated virus stability assessment during formulation development

Microbiology Spectrum
Formulation development for gene therapy viral vectors requires high-performance analytical methods that indicate or predict virus stability. Additionally, if testing involves multiple excipients, sample throughput must be sufficiently high, and the ...
Carina Rodenstein, Eva Schmid, Natalia Markova, Andreas Seidl   +3 more
doaj   +1 more source

The state of the art of adeno-associated virus-based vectors in gene therapy

Virology Journal, 2007
The adeno-associated virus (AAV) has rapidly gained popularity in gene therapy since the establishment of the first AAV2 infectious clone, in 1982, due to some of their distinguishing characteristics such as lack of pathogenicity, wide range of ...
Nardi Nance, Coura Renata
doaj   +1 more source

List of cellular proteins identified using GeLC-MS analysis of adeno-associated virus vectors.

, 2014
List of cellular proteins identified using GeLC-MS analysis of adeno-associated virus vectors.
Lingxia Chen (519218), Philipp Kapranov (13711), Bernd Hauck (519220), Hoi Yee Chow (238308), Wenman Wu (519219), Biao Dong (519216), Weidong Xiao (466400), Fraser Wright (519221), Xunbao Duan (519217), Hui Lu (12370)   +9 more
core   +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

ApoE gene therapy to treat hyperlipidemia and atherosclerosis [PDF]

, 2006
Atherosclerosis is the leading cause of death in industrialized countries and is becoming an increasingly worldwide risk to health. Apolipoprotein E (ApoE) is a blood circulating protein with pleiotropic atheroprotective properties that has emerged as a ...
Owen, JS, Evans, V, Harris, JD, Evans, Vanessa C   +3 more
core  

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