Results 81 to 90 of about 9,370,992 (242)

SerpinA3 is an Endogenous TGF‐β Receptor Antagonist that Attenuates Cardiac Fibroblast Activation and Fibrotic Remodeling

Advanced Science, EarlyView.
SerpinA3 acts as an endogenous TGF‐β receptor antagonist that binds to the extracellular domain of TGFR‐1, thereby preventing TGFR‐1–TGFR‐2 complex formation. This receptor‐level blockade suppresses TGF‐β/Smad2/3 signaling, attenuates cardiac fibroblast activation, and extracellular matrix deposition, and ultimately alleviates pressure overload–induced
Hui Wang, Jieyu Cui, Chunlu Huang, Wanyi Guo, Tong Liu, Yi Zhu, Shuai Shao, Kangyin Chen, Jinlong He, Qiankun Bao   +9 more
wiley   +1 more source

High levels of AAV vector integration into CRISPR-induced DNA breaks

Nature Communications, 2019
In-depth characterization of adeno-associated virus (AAV)-mediated CRISPR delivery is still lacking. Here, the authors show high levels of integration into Cas9-induced double-strand breaks (DSBs) in therapeutically relevant genes in vivo.
Killian S. Hanlon, Benjamin P. Kleinstiver, Sara P. Garcia, Mikołaj P. Zaborowski, Adrienn Volak, Stefan E. Spirig, Alissa Muller, Alexander A. Sousa, Shengdar Q. Tsai, Niclas E. Bengtsson, Camilla Lööv, Martin Ingelsson, Jeffrey S. Chamberlain, David P. Corey, Martin J. Aryee, J. Keith Joung, Xandra O. Breakefield, Casey A. Maguire, Bence György   +18 more
doaj   +1 more source

An RNA‐Binding Protein RBMS1 Promotes Endothelial Cell Autophagy to Maintain Vascular Homeostasis and Suppress Deep Vein Thrombosis

Advanced Science, EarlyView.
RBMS1, an RNA‐binding protein, restores vascular endothelial homeostasis by enhancing ATG3‐mediated autophagy, thereby attenuating thrombus formation and suggesting a potential avenue for further therapeutic exploration in DVT. ABSTRACT Deep vein thrombosis (DVT) is a prevalent vascular disorder characterized by aberrant coagulation within the deep ...
Chu Chu, Xinkui Liu, Nannan Fan, Xiaoyan Yu, Qiaoqiao Han, Yunhong Zhang, Wei Li, Yongqing Cai, Miaomiao Zhou, Fang Li, Feifei Shi, Zhen Zhang, Bin Wang, Xia Li   +13 more
wiley   +1 more source

AAVR-Displaying Interfaces: Serotype-Independent Adeno-Associated Virus Capture and Local Delivery Systems

Molecular Therapy: Nucleic Acids, 2019
Interfacing gene delivery vehicles with biomaterials has the potential to play a key role in diversifying gene transfer capabilities, including localized, patterned, and controlled delivery. However, strategies for modifying biomaterials to interact with
Seung-Hyun Kim, Slgirim Lee, Heehyung Lee, Mira Cho, David V. Schaffer, Jae-Hyung Jang   +5 more
doaj   +1 more source

ACSL4‐Dependent Lysosomal Lipid Peroxidation Links WTAP‐Mediated m6A Modification to Intervertebral Disc Degeneration

Advanced Science, EarlyView.
IL‐1β upregulates the protein level of WTAP, which promotes the m6A modification of ACSL4 mRNA in an IGF2BP2‐dependent manner, thereby enhancing its stability. The increased ACSL4 drives lipid peroxidation, leading to lysosomal membrane permeabilization (LMP) and impaired mitophagy, which collectively accelerate intervertebral disc degeneration (IVDD).
Shu Jia, Xu Gao, Laimin Zhu, Haixia Song, Jing Wu, Shang Chen, Sheng Gao, Lu Zhang, Chunyang Meng   +8 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
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Adeno-Associated Virus Biology

, 2011
Adeno-associated virus (AAV) was first discovered as a contaminant of adenovirus stocks in the 1960s. The development of recombinant AAV vectors (rAAV) was facilitated by early studies that generated infectious molecular clones, determined the sequence ...
Linden, R Michael, R. Michael Linden, Weitzman, Matthew D, Matthew D. Weitzman   +3 more
core   +1 more source

Early Spatial and Contextual Coding Deficits in Hippocampal CA1 Precede Performance Decline in an Alzheimer's Disease Model

Advanced Science, EarlyView.
Two‐photon calcium imaging in behaving 5xFAD mice reveals early CA1 coding deficits before behavioral decline. Task‐related place cell activity and splitter cell trajectory coding are impaired, with reduced retrospective dominance and greater prospective weighting.
Yimei Li, Mary Ann Go, Hualong Zhang, Robertas Aleksynas, Juliana Kim, Mingyang Gao, Seigfred Prado, Magdalena Sastre, Simon R. Schultz   +8 more
wiley   +1 more source

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

Molecular Therapy: Methods & Clinical Development, 2018
Recombinant adeno-associated virus (rAAV)-based gene therapy has entered a phase of clinical translation and commercialization. Despite this progress, vector integrity following production is often overlooked.
Phillip W.L. Tai, Jun Xie, Kaiyuen Fong, Matthew Seetin, Cheryl Heiner, Qin Su, Michael Weiand, Daniella Wilmot, Maria L. Zapp, Guangping Gao   +9 more
doaj   +1 more source

Viral vector-mediated RNA interference in the retina [PDF]

, 2010
RNA interference (RNAi) is a highly conserved post-transcriptional gene silencing process triggered by double-stranded RNA (dsRNA) in eukaryotic cells.
Georgiadis, A.
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