Results 111 to 120 of about 115,653 (351)

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

Seipin‐Mediated Lipid Droplet Formation in Cardiomyocytes Ameliorates Cardiac Ischemia/Reperfusion Injury

Advanced Science, EarlyView.
Lipid droplets (LDs) protect the heart against lipotoxicity in cardiac ischemia/reperfusion (I/R) injury; however, they are insufficient to prevent cardiomyocyte death. Seipin plays a central role in the insufficient formation of LDs, subsequent lipotoxicity, and myocardial injury during cardiac I/R injury.
Changyun Liu, Junxia Zhang, Yusi Chen, Geng Shen, Yufei Han, Zihao Zhou, Jinxuan Chen, Xuya Kang, Huilin Qu, Jiaxin Duanmu, Haibao Shang, Yingjia Li, Wei Huang, Yan Zhang   +13 more
wiley   +1 more source

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

Phosphorylation of the Adeno-Associated Virus Replication Proteins

Virology, 1997
The adeno-associated virus (AAV) replication proteins Rep78 and Rep68 regulate viral gene expression and DNA amplification. Their effects on both processes suggest that they play roles in all phases of the virus life cycle. We have investigated Rep protein phosphorylation to determine if this modification might alter Rep function. All four Rep proteins
Collaco, Roy, Prasad, Konkal-Matt Renuka, Trempe, James P.   +2 more
openaire   +2 more sources

CHB‐Induced Immune Zonation Chaos Elicited LXRα‐mediated Lipid Metabolism Disorders in Kupffer Cells to Induce Cancer Stem Cell Formation

Advanced Science, EarlyView.
By profiling the spatiotemporal hepatic landscape of CHB mouse models, the originally peri‐portal localized KCs migrated to the peri‐central in a CXCL9‐CXCR3‐dependent manner, facilitating their interaction with HBV+ hepatocytes. The interaction promoted LMD in KCs through ASGR1‐induced LXRα degradation, which, in turn, induced CSC formation via Stat3 ...
Jingqi Shi, Qingyu Li, Jian Li, Jian Bai, Ji Xi, Qi He, Jianglin Zhou, Xuejun Wang, Xiang Song, Xiaoju Li, Xiangpei Yue, Xiaochang Zhang, Zhen Sun, Jiangbo Li, Wen Yang, Yuke Cui, Wenjie Shu, Liang Guo, Shengqi Wang   +18 more
wiley   +1 more source

Skeletal Muscle HSF1 Alleviates Age‐Associated Sarcopenia and Mitochondrial Function Decline via SIRT3‐PGC1α Axis

Advanced Science, EarlyView.
Aged HSF1 muscle‐specific knockout mice show deteriorated muscle atrophy and metabolic dysfunction, while active HSF1 overexpression improves muscle function via activating SIRT3 to deacetylate both PGC1α1 and PGC1α4, which boosts mitochondrial function and muscle hypertrophy in a fiber‐type specific manner, and induces FNDC5/Irisin for tissue ...
Jun Zhang, Min Hu, Xia Wu, Mingwei Guo, Ying Ma, Jin Qiu, Siqi Wang, Yuxiang Cao, Yinzhao Zhong, Fangfang Chen, Yiwen Wang, Wei Wei, Yan Lu, Yong Zhang, Junjie Xiao, Zhenji Gan, Cheng Hu, Xinran Ma, Lingyan Xu   +18 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

Gene Therapy Rescues Cardiac Dysfunction in Duchenne Muscular Dystrophy Mice by Elevating Cardiomyocyte Deoxy-Adenosine Triphosphate

JACC: Basic to Translational Science, 2019
Summary: Mutations in the gene encoding for dystrophin leads to structural and functional deterioration of cardiomyocytes and is a hallmark of cardiomyopathy in Duchenne muscular dystrophy (DMD) patients.
Stephen C. Kolwicz, Jr., PhD, John K. Hall, PhD, Farid Moussavi-Harami, MD, Xiolan Chen, PhD, Stephen D. Hauschka, PhD, Jeffrey S. Chamberlain, PhD, Michael Regnier, PhD, Guy L. Odom, PhD   +7 more
doaj   +1 more source

Astrocytic PERK Deficiency Drives Prefrontal Circuit Dysfunction and Depressive‐Like Behaviors

Advanced Science, EarlyView.
Chen et al. show that the endoplasmic reticulum (ER) stress sensor PERK is downregulated in prefrontal cortex (PFC) astrocytes in major depressive disorder and in chronic‐stress mouse models. In young mice, astrocyte‐specific PERK loss reduces the synaptogenic cue thrombospondin‐1 (TSP1), leading to synaptic and circuit deficits and depressive‐like ...
Kai Chen, Riya Gupta, Yosuke M. Morizawa, Yu Qin, Xingyu Du, Cynthia Pang, Osama Al‐Dalahmah, Maura B. Dupont, Guang Yang   +8 more
wiley   +1 more source

Viral Vector-based Improvement of Optic Nerve Regeneration: Characterization of Individual Axons\u27 Growth Patterns and Synaptogenesis in a Visual Target [PDF]

, 2015
Lack of axon growth ability in the central nervous system poses a major barrier to achieving functional connectivity after injury. Thus, a non-transgenic regenerative approach to reinnervating targets has important implications in clinical and research ...
Blackmore, Murray G., Luo, Xueting, Salgueiro, Yadira, Yungher, Benjamin J.   +3 more
core   +1 more source