Results 61 to 70 of about 73,809 (216)

Comprehensive mutagenesis maps the effect of all single-codon mutations in the AAV2 rep gene on AAV production

eLife
Recombinant adeno-associated viruses (rAAVs) are the predominant gene therapy vector. Several rAAV vectored therapies have achieved regulatory approval, but production of sufficient rAAV quantities remains difficult.
Nina K Jain, Pierce J Ogden, George M Church   +2 more
doaj   +1 more source

A Blood‐Derived Factor Rescues ALS: Platelet Factor 4 Activates OPTN‐Dependent Autophagy to Clear SOD1 Aggregates Independently of PINK1

Advanced Science, EarlyView.
Systemic platelet factor 4 (PF4) is significantly depleted in amyotrophic lateral sclerosis (ALS). Peripheral PF4 replenishment restores central proteostasis by driving OPTN‐dependent, PINK1‐independent selective autophagy in motor neurons. This intervention effectively clears toxic SOD1 aggregates, blunts glial activation, and preserves neuromuscular ...
Qingjian Xie, Yanbo Zhu, Wenhua Jiang, Haobo Xie, Yaojia Li, Mengjie Hu, Kezheng Li, Huan Yu, Yixuan Pan, Chaoyi Jiang, Xueqin Song, Dongsheng Fan, Binbin Deng   +12 more
wiley   +1 more source

Association between adeno‐associated virus genomic titers and intracellular plasmid levels

Engineering Reports
The recombinant adeno‐associated viral (rAAV) vector is one of the most effective viral vectors in gene therapy because of its low immunogenicity, high transduction efficiency, broad tissue specificity, and long‐term transgene expression ability. HEK293T
Kyoko Masumi‐Koizumi, Yuzhe Yuan, Kiyoko Higashiyama, Keisuke Yusa, Kazuhisa Uchida   +4 more
doaj   +1 more source

Targeting the KCNC3‐Dependent Anterior Insular‐Lateral Orbitofrontal Cortex Glutamatergic Circuit Attenuates Post‐Traumatic Anxiety

Advanced Science, EarlyView.
Frontal lobe traumatic brain injury is associated with hyperactivity of an insular–orbitofrontal circuit in both patients and mice. By combination of integrating functional imaging, cell‐type–specific circuit manipulation, single‐cell transcriptomics, and whole‐cell recordings, this work identifies the downregulation of the potassium channel KCNC3 in ...
Meng‐Ge Li, Xiao‐Bo Qian, Hai‐Long Zhang, Di Li, Ling Ji, Xin‐Chun Xu, Jia‐Sheng Ding, Li‐Jun Yin, Li Zhang, Guang‐Yin Xu, Rong Gao   +10 more
wiley   +1 more source

YAP1‐Driven Pathogenic Fibro‐Adipogenic Progenitors Secrete IL‐6 and FGF21 to Mediate Muscle‐Bone Crosstalk and Promote Bone Loss

Advanced Science, EarlyView.
Fibro‐adipogenic progenitors (FAPs) in atrophic muscle undergo YAP1‐driven pathogenic activation, secreting IL‐6 and FGF21 as bone‐catabolic myokines that mediate muscle‐bone crosstalk and promote bone loss. Genetic or pharmacological targeting of this YAP1‐FAP‐myokine axis rescues skeletal deterioration, identifying FAP‐derived myokines as therapeutic
Xiaoyu Cai, Tao Xu, Rui Ma, Weikun Meng, Shutao Gao, Yakefu Abulizi, Alafate Kahaer, Chuanhui Xun, Maierdan Maimaiti, Rui Cao, Weidong Liang, Polati Maimaiti, Qiang Deng, Jun Sheng, Ting Wang, Mingchong Liu, Weibin Sheng   +16 more
wiley   +1 more source

Gαi1/3 Are Essential for BMP2‐Induced Osteogenesis by Bridging BMP Receptors to the Gab1 Signaling Complex

Advanced Science, EarlyView.
The Gαi1/3 protein is a crucial connecting protein closely related to the signal transduction process of bone formation‐related cytokines such as BMP2. Gαi1/3 mediates downstream signaling pathways such as Smads and MAPK by binding to adaptor proteins like Gab1, thereby promoting the osteogenic effect of BMSCs.
Huajian Shan, Chaowen Bai, Yifei Zhu, Chenyang Xu, Sonngtao Li, Qize Sun, Chang She, Jinyu Bai, Cong Cao, Yitian Yang, Xiaozhong Zhou   +10 more
wiley   +1 more source

Size does matter: overcoming the adeno-associated virus packaging limit

Respiratory Research, 2000
Recombinant adeno-associated virus (rAAV) vectors mediate long-term gene transfer without any known toxicity. The primary limitation of rAAV has been the small size of the virion (20 nm), which only permits the packaging of 4.7 kilobases (kb) of ...
Flotte Terence R
doaj   +1 more source

USP5 Stabilizes TGFBR1 to Drive Vascular Smooth Muscle Cell Senescence and Atherosclerosis

Advanced Science, EarlyView.
This study reveals that USP5 drives vascular smooth muscle cell senescence and atherosclerosis by stabilizing TGFBR1, suppressing IDH2, and promoting glycolytic reprogramming, identifying the USP5‐TGFBR1‐IDH2 axis as a potential therapeutic target. ABSTRACT Vascular smooth muscle cell (VSMC) senescence contributes importantly to atherosclerotic plaque ...
Xinhai Cui, Yuanlong Hu, Lin Lin, Lei Zhang, Chao Li, Yunlun Li   +5 more
wiley   +1 more source

Glycosylation of recombinant adeno-associated virus serotype 6

Molecular Therapy: Methods & Clinical Development
Glycosylation of biopharmaceuticals can affect their safety and efficacy. Glycans can occur on recombinant adeno-associated viruses (rAAVs) that are used for gene therapy; however, the types of glycans that attach to rAAVs are controversial.
Yuki Yamaguchi, Kentaro Ishii, Sachiko Koizumi, Hiroaki Sakaue, Takahiro Maruno, Mitsuko Fukuhara, Risa Shibuya, Yasuo Tsunaka, Aoba Matsushita, Karin Bandoh, Tetsuo Torisu, Chie Murata-Kishimoto, Azusa Tomioka, Saho Mizukado, Hiroyuki Kaji, Yuji Kashiwakura, Tsukasa Ohmori, Atsushi Kuno, Susumu Uchiyama   +18 more
doaj   +1 more source

Systematic Multi‐Level Analyses Decode the Arthritis‐Neurodegeneration Axis With In Vivo Validation

Advanced Science, EarlyView.
Arthritis and neurodegeneration are usually studied as separate disorders, but this study connects them through population evidence, genetic inference, transcriptomic mapping, and mouse models. It highlights RNF40 as a context‐dependent joint‐brain candidate, induced in inflammatory joints yet functionally linked to dopamine‐neuron vulnerability ...
Jinwen Wang, Wenhui Xie, Lei Yang, Zhengrong Wang, Yi Huang, Peng Huang, Yuan Liu, Jun Hu   +7 more
wiley   +1 more source