Results 71 to 80 of about 115,653 (351)

Potential for cellular stress response to hepatic factor VIII expression from AAV vector

Molecular Therapy: Methods & Clinical Development, 2016
Hemophilia A and B are coagulation disorders resulting from the loss of functional coagulation factor VIII (FVIII) or factor IX proteins, respectively.
Irene Zolotukhin, David M Markusic, Brett Palaschak, Brad E Hoffman, Meera A Srikanthan, Roland W Herzog   +5 more
doaj   +1 more source

The Adeno-Associated Virus Genome Packaging Puzzle [PDF]

Journal of Molecular and Genetic Medicine, 2015
Chen Ling1,2, Yuan Wang1-3, Yuan Lu4, Lina Wang1-3, Giridhara R Jayandharan5, George V Aslanidi1,2, Baozheng Li1,2, Binbin Cheng1,3, Wenqin Ma1,2, Thomas Lentz6, Changquan Ling3, Xiao Xiao6,7, R Jude Samulski6, Nicholas Muzyczka2,8,9 and Arun Srivastava1,2,8-10* 1Division of Cellular and Molecular Therapy, Department of Pediatrics, University of ...
Chen, Ling, Yuan, Wang, Yuan, Lu, Lina, Wang, Giridhara R, Jayandharan, George V, Aslanidi, Baozheng, Li, Binbin, Cheng, Wenqin, Ma, Thomas, Lentz, Changquan, Ling, Xiao, Xiao, R Jude, Samulski, Nicholas, Muzyczka, Arun, Srivastava   +14 more
openaire   +2 more sources

Inhibiting Liver‐Derived C3 Protein Rescues Anesthesia/Surgery‐Induced Cognitive Impairment, Synaptic Disorders, and Microglial Phagocytosis

Advanced Science, EarlyView.
This study provides evidence that anesthesia/surgery can induce the BBB breakdown and promote the release of hepatogenous C3 protein into the blood. This surgical dual factors ultimately drove C3 to cross the damaged BBB and selectively colocalize with C3aR in the hippocampus, which results in structural and functional injury of synapse, C3aR‐mediated ...
Qianqian Wu, Peilin Cong, Zhouxiang Li, Yuxin Zhang, Huanghui Wu, Qian Zhang, Yawei Li, Li Tian, Qingyuan Miao, Yinggang Zheng, Hui Zhang, Qian Chen, Enduo Feng, Xinyang Li, Zheping Chen, Dong‐Xin Wang, Xinwei Huang, Lize Xiong   +17 more
wiley   +1 more source

Circular RNA PTPN4 Contributes to Blood‐Brain Barrier Disruption during Early Epileptogenesis

Advanced Science, EarlyView.
Epileptic condition induces CircPTPN4 upregulation, which promotes ECE‐1 expression through competitive sequestration of miR‐145a‐5p. The elevated ECE‐1 catalyzes the ET‐1 production, leading to p38/MAPK pathway activation and subsequent downregulation of tight junction protein expression. This cascade results in increased BBB permeability and enhanced
Jiurong Yang, Yang Hu, Feiyu Wang, Xintao Peng, Honggang Qi, Yuanyuan Yao, Canyu Zhang, Lijie Zhou, Xuemei Liang, Kang Xu, Cong Zhang, Aifeng Zhang, Chen Chen, Yu Zeng, Chenchen Zhang, Guangming Gan, Xinjian Zhu   +16 more
wiley   +1 more source

Mucopolysaccharidosis type IIIB: a current review and exploration of the AAV therapy landscape

Neural Regeneration Research
Mucopolysaccharidoses type IIIB is a rare genetic disorder caused by mutations in the gene that encodes for N-acetyl-alpha-glucosaminidase. This results in the aggregation of heparan sulfate polysaccharides within cell lysosomes that leads to progressive
Courtney J Rouse, Victoria N Jensen, Coy D Heldermon   +2 more
doaj   +1 more source

Gene Transfer of Engineered Calmodulin Alleviates Ventricular Arrhythmias in a Calsequestrin-Associated Mouse Model of Catecholaminergic Polymorphic Ventricular Tachycardia [PDF]

, 2018
Catecholaminergic polymorphic ventricular tachycardia (CPVT) is a familial arrhythmogenic syndrome characterized by sudden death. There are several genetic forms of CPVT associated with mutations in genes encoding the cardiac ryanodine receptor (RyR2 ...
Belevych, Andriy E, Bonilla, Ingrid, Davis, Jonathan P, Gy\uf6rke, S\ue1ndor, Ho, Hsiang-Ting, Knollmann, Bjorn C, Liu, Bin, Priori, Silvia G, Radwa\u144ski, Przemys\u142aw B, Shettigar, Vikram, Tikunova, Svetlana B, Volpe, Pompeo, Walton, Shane D   +12 more
core   +1 more source

Realgar‐Induced CNS Toxicity: Exploring OTC‐Mediated Ornithine Regulation of ZBTB7A Inhibits Astrocyte Glycolysis Based on the Liver–Brain Axis

Advanced Science, EarlyView.
Arsenic in realgar induces CNS toxicity through direct and indirect effects: it activates ZBTB7A in astrocytes, which in turn inhibits glycolysis and impairs mice's learning, memory, and exploration; it also suppresses OTC in the liver to block the ornithine cycle, leading to blood ornithine accumulation that crosses into the frontal lobe and further ...
Ping Ye, Zhen Li, Huanyong Fu, Shuai Wang, Xingang Cui, Chengbo Song, Chao Ma, Hong Jiang   +7 more
wiley   +1 more source

Adeno-associated virus for cystic fibrosis gene therapy

Brazilian Journal of Medical and Biological Research, 2011
Gene therapy is an alternative treatment for genetic lung disease, especially monogenic disorders such as cystic fibrosis. Cystic fibrosis is a severe autosomal recessive disease affecting one in 2500 live births in the white population, caused by ...
S.V. Martini, P.R.M. Rocco, M.M. Morales
doaj   +3 more sources

Microglial Deletion of Hrh4 Alleviates Alzheimer's Disease Pathologies by Enhancing Microglial Phagocytosis of Amyloid‐β and Tau

Advanced Science, EarlyView.
Histamine H4 receptor (H4R) antagonist VUF6002 mimics low‐dose X‐ray irradiation in aged Alzheimer's disease (AD) mice, enhancing microglial clearance of amyloid‐beta/hyperphosphorylated tau aggregates and restoring cognition. Microglial H4R deletion activates cAMP/TGF‐β1/Smad3 pathway, enhancing phagocytosis, while TGF‐β receptor 1 deletion abolishes ...
Yi‐Jun Xu, Tan Wu, Larry Tso‐Lun Lo, Chi‐Chiu Ko, Xin Wang, Chi Him Eddie Ma   +5 more
wiley   +1 more source

Viral Hybrid Vectors for Somatic Integration - Are They the Better Solution? [PDF]

, 2009
The turbulent history of clinical trials in viral gene therapy has taught us important lessons about vector design and safety issues. Much effort was spent on analyzing genotoxicity after somatic integration of therapeutic DNA into the host genome. Based
Ehrhardt, Anja, Müther, Nadine, Noske, Nadja   +2 more
core   +4 more sources