Results 51 to 60 of about 110,545 (302)

Respiratory Organ‐on‐a‐Chip for Disease Modeling: From Architecture to Functional Integration

Advanced Healthcare Materials, EarlyView.
Respiratory organ‐on‐a‐chip (ROC) models capture key mechanical and cellular cues of the human respiratory system, enabling quantitative dissection of disease mechanisms. This review links ROC architectures to disease modeling, functional integration, and commercialization, and proposes a decision framework that aligns model complexity with mechanistic
Jinzhuo Hu, Yongjie Tang, Sidi Liu, Tao Xu, Qian Liu   +4 more
wiley   +1 more source

Adeno-Associated Virus VP1u Exhibits Protease Activity

Viruses, 2019
Adeno-associated viruses (AAVs) are being developed for gene delivery applications, with more than 100 ongoing clinical trials aimed at the treatment of monogenic diseases.
Justin J. Kurian, Renuk Lakshmanan, William M. Chmely, Joshua A. Hull, Jennifer C. Yu, Antonette Bennett, Robert McKenna, Mavis Agbandje-McKenna   +7 more
doaj   +1 more source

Functional rescue of dystrophin deficiency in mice caused by frameshift mutations using Campylobacter jejuni Cas9 [PDF]

, 2018
Duchenne muscular dystrophy (DMD) is a fatal, X-linked muscle wasting disease caused by mutations in the DMD gene. In 51% of DMD cases, a reading frame is disrupted because of deletion of several exons.
Cappellari, O, Cho, H-Y, Dickson, G, Kim, D, Kim, E, Kim, J-S, Koo, T, Lu-Nguyen, N B, Malerba, A, Popplewell, L   +9 more
core   +2 more sources

Expression Profiles of Bovine Adeno-Associated Virus and Avian Adeno-Associated Virus Display Significant Similarity to That of Adeno-Associated Virus Type 5 [PDF]

Journal of Virology, 2006
ABSTRACT We present the first detailed expression profiles of nonprimate-derived adeno-associated viruses, namely, bovine adeno-associated virus (B-AAV) and avian adeno-associated virus (A-AAV), which were obtained after the infection of cell lines derived from their natural hosts.
Jianming, Qiu, Fang, Cheng, David J, Pintel   +2 more
openaire   +2 more sources

IL‐3 Modulates Microglia Polarization and Attenuates Neuroinflammation in Traumatic Brain Injury

Advanced Science, EarlyView.
IL‐3 recruits PRDX1 through IL‐3R to modulate the inflammatory response of microglia via the KEAP1‐NRF2‐HO‐1/NF‐κB pathway to attenuate neuroinflammation in traumatic brain injury. ABSTRACT Microglia play a crucial role in the progression of neuroinflammation following traumatic brain injury (TBI). Interleukin‐3 (IL‐3), a significant regulatory factor,
Nana Huang, Qingchen Zhang, Yanrui Chen, Dapeng Yu, Ronghan Liu, Jianning Kang, Xiang Fang, Ying Zhang, Hong Bian, Yanxin Zhao, Yongcheng Yin, Ce Zhang, Yanfei Jia, Qingfa Chen, Yuepeng Fang, Shang Li, Fang Li, Zhengxin Jin, Bin Ning   +18 more
wiley   +1 more source

Pharmacology of Recombinant Adeno-associated Virus Production

Molecular Therapy: Methods & Clinical Development, 2018
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases.
Magalie Penaud-Budloo, Achille François, Nathalie Clément, Eduard Ayuso   +3 more
doaj   +1 more source

Suprachoroidal and Subretinal Injections of AAV Using Transscleral Microneedles for Retinal Gene Delivery in Nonhuman Primates. [PDF]

, 2020
Retinal gene therapy using adeno-associated viruses (AAVs) is constrained by the mode of viral vector delivery. Intravitreal AAV injections are impeded by the internal limiting membrane barrier, while subretinal injections require invasive surgery and ...
Chung, Sook Hyun, Mollhoff, Iris N, Nguyen, Uyen Tu, Noronha, Glenn, Taraborelli, Donna, Thomasy, Sara M, Yiu, Glenn, Yoo, Jesse   +7 more
core  

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

Piezo2 Mediates a Vicious Cycle of “Mechanical Homeostasis Imbalance—Inflammation” in Sensory Nerves and the Cartilage Endplate

Advanced Science, EarlyView.
In a lumbar spine instability model, dorsal root ganglion cells mediate the perception of relevant mechanical stresses through Piezo2 and subsequently release CGRP. CGRP activates the NF‐κB signaling pathway in cartilage endplate cells through the receptor RAMP1.
Hanpeng Xu, Wen Geng, Zhi Du, Yifan Du, Di Wu, Bide Tong, Huaizhen Liang, Xingyu Zhou, Zixuan Ou, Junyu Wei, Kun Wang, Yu Song, Wenbin Hua, Yan Xu, Wencan Ke, Bingjin Wang, Cao Yang   +16 more
wiley   +1 more source

Extracellular vesicles enhance pulmonary transduction of stably associated adeno‐associated virus following intratracheal administration

Journal of Extracellular Vesicles, 2023
Adeno‐associated virus (AAV) vector has shown multiple clinical breakthroughs, but its clinical implementation in inhaled gene therapy remains elusive due to difficulty in transducing lung airway cells.
Gijung Kwak, Olesia Gololobova, Neeraj Sharma, Colin Caine, Marina Mazur, Kathleen Mulka, Natalie E. West, George M. Solomon, Garry R. Cutting, Kenneth W. Witwer, Steven M. Rowe, Michael Paulaitis, George Aslanidi, Jung Soo Suk   +13 more
doaj   +1 more source