Results 131 to 140 of about 361,542 (380)

Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

Molecular Therapy, 2009
Therapeutic gene delivery to the whole spinal cord is a major challenge for the treatment of motor neuron (MN) diseases. Systemic administration of viral gene vectors would provide an optimal means for the long-term delivery of therapeutic molecules from
S. Duqué, B. Joussemet, C. Rivière, Thibaut Marais, L. Dubreil, A. Douar, J. Fyfe, P. Moullier, M. Colle, M. Barkats   +9 more
semanticscholar   +1 more source

Progress in the use of adeno-associated viral vectors for gene therapy [PDF]

, 2004
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Braun-Falco, M., Buning, H., Hallek, M.
core   +1 more source

Chloroplast Stress Signals Orchestrate Epidermis‐Specific Remodeling of Mitochondria and ER Under High Light

Advanced Science, EarlyView.
High light exposure triggers an epidermis‐specific remodeling of mitochondria and ER in Arabidopsis, driven by chloroplast‐derived signals. Live‐cell imaging shows that HL rapidly suppresses mitochondrial motility, followed by fusion‐driven elongation and ER cisternal expansion.
Evan R. Angelos, Hee‐Seung Choi, Jingzhe Guo, Andrea A. Zanini, Tessa M. Burch‐Smith, Emily Snyder, Matthew Part, Wilhelmina van de Ven, Manhoi Hur, Gerd Ulrich Balcke, Alain Tissier, Quanqing Zhang, Katayoon Dehesh   +12 more
wiley   +1 more source

Vaccinia-based oncolytic immunotherapy Pexastimogene Devacirepvec in patients with advanced hepatocellular carcinoma after sorafenib failure: a randomized multicenter Phase IIb trial (TRAVERSE)

OncoImmunology, 2019
Pexastimogene devacirepvec (Pexa-Vec) is a vaccinia virus-based oncolytic immunotherapy designed to preferentially replicate in and destroy tumor cells while stimulating anti-tumor immunity by expressing GM-CSF.
M. Moehler, J. Heo, H.C. Lee, W.Y. Tak, Y. Chao, S.W. Paik, H.J. Yim, K.S. Byun, A. Baron, G. Ungerechts, D. Jonker, L. Ruo, M. Cho, A. Kaubisch, H. Wege, P. Merle, O. Ebert, F. Habersetzer, J.F. Blanc, Olivier Rosmorduc, R. Lencioni, R. Patt, A.M. Leen, F. Foerster, M. Homerin, N. Stojkowitz, M. Lusky, J.M. Limacher, M. Hennequi, N. Gaspar, B. McFadden, N. De Silva, D. Shen, A. Pelusio, D.H. Kirn, C.J. Breitbach, J.M. Burke   +36 more
doaj   +1 more source

Spontaneous atopic dermatitis in mice expressing an inducible thymic stromal lymphopoietin transgene specifically in the skin

Journal of Experimental Medicine, 2005
The cytokine thymic stromal lymphopoietin (TSLP) has recently been implicated in the pathogenesis of atopic dermatitis (AD) and other allergic diseases in humans.
Jane Yoo, M. Omori, Dora Gyarmati, Baohua Zhou, Theingi Aye, A. Brewer, M. Comeau, D. Campbell, S. Ziegler   +8 more
semanticscholar   +1 more source

A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice. [PDF]

, 2001
Dystrophin-deficient muscles experience large reductions in expression of nitric oxide synthase (NOS), which suggests that NO deficiency may influence the dystrophic pathology.
Spencer, MJ, Tidball, JG, Wehling, M
core  

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]

, 2003
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM, Nathwani, AC, Ng, CYC, Spence, Y, Zhou, J   +4 more
core   +1 more source

Protease‐Activated Plasmonic Nanosensors for Predictive Ultrasound‐Guided Photoacoustic Imaging of Tumor Responses to Adoptive T Cell Therapy

Advanced Science, EarlyView.
Protease‐activated plasmonic nanosensors are demonstrated to noninvasively monitor antitumor T cell activity in tumors with anatomical information upon adoptive T cell transfer via ultrasound‐guided photoacoustic imaging. These nanosensors are actuated by granzyme B, a protease secreted by activated cytotoxic T cells during tumor cell killing, which ...
Myeongsoo Kim, Seoyoon Song, Ali Zamat, Paul S. Pelkowski, Shivashankar Subramanian, Melissa Cadena, Sydney Fabrega, Meredith Brienen, Jinhwan Kim, Gabriel A. Kwong, Stanislav Y. Emelianov   +10 more
wiley   +1 more source

Targeting DNA‐LNPs to Endothelial Cells Improves Expression Magnitude, Duration, and Specificity

Advanced Science, EarlyView.
Attaching antibodies against endothelial cell surface proteins redirects the delivery and expression of DNA‐lipid nanoparticles to organs of interest. Our targeted nanoparticles enable organ‐selective DNA expression in the endothelium of the lungs, brain, or spleen, providing a therapeutic platform for dozens of endothelial‐centric diseases.
Nicolas Marzolini, Taylor V. Brysgel, Ryan J. Rahman, Eno‐Obong Essien, Saw Y. Nwe, Jichuan Wu, Aparajeeta Majumder, Manthan N. Patel, Sachchidanand Tiwari, Carolann L. Espy, Fengyi Dong, Sabrina A. Santos‐De León, Anit Shah, Vladimir V. Shuvaev, Elizabeth D. Hood, Liam S. Chase, Drew Weissman, Jeremy B. Katzen, David B. Frank, Mariko L. Bennett, Oscar A. Marcos‐Contreras, Jacob W. Myerson, Vladimir R. Muzykantov, Sahily Reyes‐Esteves, Jacob S. Brenner   +24 more
wiley   +1 more source

Intestinal peroxisome proliferator‐activated receptor α‐fatty acid‐binding protein 1 axis modulates nonalcoholic steatohepatitis

Hepatology, EarlyView., 2022
Abstract Background and Aims Peroxisome proliferator‐activated receptor α (PPARα) regulates fatty acid transport and catabolism in liver. However, the role of intestinal PPARα in lipid homeostasis is largely unknown. Here, intestinal PPARα was examined for its modulation of obesity and NASH. Approach and Results Intestinal PPARα was activated and fatty
Tingting Yan, Yuhong Luo, Nana Yan, Keisuke Hamada, Nan Zhao, Yangliu Xia, Ping Wang, Changdong Zhao, Dan Qi, Shoumei Yang, Lulu Sun, Jie Cai, Qiong Wang, Changtao Jiang, Oksana Gavrilova, Kristopher W. Krausz, Daxesh P. Patel, Xiaoting Yu, Xuan Wu, Haiping Hao, Weiwei Liu, Aijuan Qu, Frank J. Gonzalez   +22 more
wiley   +1 more source