Results 111 to 120 of about 116,785 (352)

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

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

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 ...
Davidoff, AM, Hanawa, H, Hoffer, FA, Hu, Y, Lozier, JN, Mandrell, TD, Nathwani, AC, Ng, CYC, Nienhuis, AW, Nikanorov, A, Slaughter, C, Vanin, EF, Zhou, J   +12 more
core   +1 more source

OTUD6A in Airway Epithelial Cells Exacerbates Allergic Asthma by Promoting Airway Inflammation and Airway Remodeling Through Deubiquitination of hResistin/mRELMα

Advanced Science, EarlyView.
This study elucidates a novel mechanistic role of the deubiquitinase OTUD6A in asthma pathogenesis, uncovering its regulatory function in airway inflammation and airway remodeling through the stabilization of hResistin/mRELMα. This study offers a novel regulatory axis (OTUD6A‐hResistin/mRELMα) in asthma pathogenesis and OTUD6A inhibition as a potential
Weiting Pan, Xinru Xi, Wei Dai, Tingfang Xiao, Yeqing Chen, Xuanyu Chen, Xiangting Ge, Chengguang Zhao, Hui Zhang, Yali Zhang, Weixi Zhang   +10 more
wiley   +1 more source

An ANGPTL4-ceramide-protein kinase Cζ axis mediates chronic glucocorticoid exposure-induced hepatic steatosis and hypertriglyceridemia in mice. [PDF]

, 2019
Chronic or excess glucocorticoid exposure causes lipid disorders such as hypertriglyceridemia and hepatic steatosis. Angptl4 (angiopoietin-like 4), a primary target gene of the glucocorticoid receptor in hepatocytes and adipocytes, is required for ...
Cheang, Rachel T, Chen, Tzu-Chieh, Fitch, Mark D, Gray, Nora E, Hellerstein, Marc K, Kanamaluru, Deepthi, Lee, Justin Y, Lee, Rebecca A, Tan, Jenna H, Tsai, Sam L, Wang, Jen-Chywan, Yiv, Nicholas   +11 more
core  

TP53BP2 Promotes Placental Autophagy and Preeclampsia via G9a and DNMT1 Cooperatively Modulating E2F1

Advanced Science, EarlyView.
G9a, and DNA Methyltransferase1 (DNMT1) cooperatively modulates E2F1 on the promoter of tumor suppressor p53‐binding protein 2 (TP53BP2) increased autophagy in preeclampsia. TP53BP2 promotes autophagy in trophoblasts through DNA methylation and H3K9me2‐mediated transcriptional regulation.
Nan Jiang, Shaoju Jin, Shaoying Wen, Wen Zeng, Chen Wang, Jingyu Wang, Qingyun Song, Guizhong Li, Pengzhi Yin, Yuhui Liao, Yuee Chai, Huiping Zhang, Shengchao Ma   +12 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 Transfer of Calcitonin Gene-Related Peptide Inhibits Macrophages and Inflammatory Mediators in Vein Graft Disease [PDF]

, 2008
Vein graft disease is a chronic inflammatory disease and limits the late results of coronary revascularization. Calcitonin gene-related peptide (CGRP) inhibits macrophages infiltrated and inflammatory mediators, we hypothesized that transfected CGRP gene
Hongsui Wu, Huanxin Yang, Jian Su, Jian Zhuang, Jianguang Chen, Shiliang Chen, Xiaoning Zhang, Yuhui Zheng, Zhihong Chen   +8 more
core   +1 more source

CRISPLD2 Attenuates Intervertebral Disc Degeneration by Suppressing Oxidative Stress‐Induced Ferroptosis through the miR‐548I‐IL17A Axis

Advanced Science, EarlyView.
This study identifies CRISPLD2 as a key protector against IVDD. By regulating ferroptosis through the CRISPLD2–miR‐548I–IL17A axis, CRISPLD2 maintains NPCs homeostasis and reduces oxidative stress. Restoring CRISPLD2 expression effectively alleviates disc degeneration and highlights a promising therapeutic strategy for discogenic low back pain ...
Yangyang Shi, Fudong Li, Tianyi Zhao, Kaiqiang Sun, Danying Zhang, Chen Yan, Ximing Xu, Jingchuang Sun, Xulin Hu, Xin Yong, Jiangang Shi, Yuan Wang   +11 more
wiley   +1 more source

Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno‐Associated Virus

Advanced Science, 2019
Delivery of genome editing tools to mammalian zygotes has revolutionized animal modeling. However, the mechanical delivery method to introduce genes and proteins to zygotes remains a challenge for some animal species that are important in biomedical ...
Dan Wang, Yuyu Niu, Lingzhi Ren, Yu Kang, Phillip W. L. Tai, Chenyang Si, Craig A. Mendonca, Hong Ma, Guangping Gao, Weizhi Ji   +9 more
doaj   +1 more source