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Molecular Therapy: the Journal of the American Society of Gene Therapy

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https://www.readbyqxmd.com/read/28082074/rescue-of-hearing-by-gene-delivery-to-inner-ear-hair-cells-using-exosome-associated-aav
#1
Bence György, Cyrille Sage, Artur A Indzhykulian, Deborah I Scheffer, Alain R Brisson, Sisareuth Tan, Xudong Wu, Adrienn Volak, Dakai Mu, Panos I Tamvakologos, Yaqiao Li, Zachary Fitzpatrick, Maria Ericsson, Xandra O Breakefield, David P Corey, Casey A Maguire
Adeno-associated virus (AAV) is a safe and effective vector for gene therapy for retinal disorders. Gene therapy for hearing disorders is not as advanced, in part because gene delivery to sensory hair cells of the inner ear is inefficient. Although AAV transduces the inner hair cells of the mouse cochlea, outer hair cells remain refractory to transduction. Here, we demonstrate that a vector, exosome-associated AAV (exo-AAV), is a potent carrier of transgenes to all inner ear hair cells. Exo-AAV1-GFP is more efficient than conventional AAV1-GFP, both in mouse cochlear explants in vitro and with direct cochlear injection in vivo...
January 9, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28089078/hucmsc-exosome-derived-gpx1-is-required-for-the-recovery-of-hepatic-oxidant-injury
#2
Yongmin Yan, Wenqian Jiang, Youwen Tan, Shengqiang Zou, Hongguang Zhang, Fei Mao, Aihua Gong, Hui Qian, Wenrong Xu
Exosomes are small biological membrane vesicles secreted by various cells, including mesenchymal stem cells (MSCs). We previously reported that MSC-derived exosomes (MSC-Ex) can elicit hepatoprotective effects against toxicant-induced injury. However, the success of MSC-Ex-based therapy for treatment of liver diseases and the underlying mechanisms have not been well characterized. We used human umbilical cord MSC-derived exosome (hucMSC-Ex) administrated by tail vein or oral gavage at different doses and, in engrafted liver mouse models, noted antioxidant and anti-apoptotic effects and rescue from liver failure...
January 6, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966563/corrigendum-to-a-self-restricted-crispr-system-to-reduce-off-target-effects
#3
(no author information available yet)
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966562/one-size-fits-all-ethical-considerations-for-examining-efficacy-in-first-in-human-pluripotent-stem-cell-studies
#4
Michelle Gjl Habets, Johannes Jm van Delden, Sophie L Niemansburg, Harold L Atkins, Annelien L Bredenoord
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966561/in-this-issue
#5
(no author information available yet)
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966560/research-highlights
#6
(no author information available yet)
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966559/corrigendum-to-targeted-delivery-of-c-ebp%C3%AE-sarna-by-pancreatic-ductal-adenocarcinoma-specific-rna-aptamers-inhibits-tumor-growth-in-vivo
#7
(no author information available yet)
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27966558/gene-therapy-for-epidermolysis-bullosa-sticky-business
#8
EDITORIAL
Alexander Nyström, Leena Bruckner-Tuderman
No abstract text is available yet for this article.
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27731313/targeted-delivery-of-an-anti-inflammatory-pde4-inhibitor-to-immune-cells-via-an-antibody-drug-conjugate
#9
Shan Yu, Aaron D Pearson, Reyna Kv Lim, David T Rodgers, Sijia Li, Holly B Parker, Meredith Weglarz, Eric N Hampton, Michael J Bollong, Jiayin Shen, Claudio Zambaldo, Danling Wang, Ashley K Woods, Timothy M Wright, Peter G Schultz, Stephanie A Kazane, Travis S Young, Matthew S Tremblay
Phosphodiesterase 4 (PDE4) inhibitors are approved for the treatment of some moderate to severe inflammatory conditions. However, dose-limiting side effects in the central nervous system and gastrointestinal tract, including nausea, emesis, headache, and diarrhea, have impeded the broader therapeutic application of PDE4 inhibitors. We sought to exploit the wealth of validation surrounding PDE4 inhibition by improving the therapeutic index through generation of an antibody-drug conjugate (ADC) that selectively targets immune cells through the CD11a antigen...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27669655/robust-oncolytic-virotherapy-induces-tumor-lysis-syndrome-and-associated-toxicities-in-the-mpc-11-plasmacytoma-model
#10
Lianwen Zhang, Michael B Steele, Nathan Jenks, Jacquelyn Grell, Marshall Behrens, Rebecca Nace, Shruthi Naik, Mark J Federspiel, Stephen J Russell, Kah-Whye Peng
Tumor-selective oncolytic vesicular stomatitis viruses (VSVs) are being evaluated in clinical trials. Here, we report that the MPC-11 murine plasmacytoma model is so extraordinarily susceptible to oncolytic VSVs that a low dose of virus leads to extensive intratumoral viral replication, sustained viremia, intravascular coagulation, and a rapidly fatal tumor lysis syndrome (TLS). Rapid softening, shrinkage and hemorrhagic necrosis of flank tumors was noted within 1-2 days after virus administration, leading to hyperkalemia, hyperphosphatemia, hypocalcemia, hyperuricemia, increase in plasma cell free DNA, lymphopenia, consumptive coagulopathy, increase in fibrinogen degradation products, decreased liver function tests, dehydration, weight loss, and euthanasia or death after 5-8 days...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27658524/low-dose-gene-therapy-reduces-the-frequency-of-enzyme-replacement-therapy-in-a-mouse-model-of-lysosomal-storage-disease
#11
Marialuisa Alliegro, Rita Ferla, Edoardo Nusco, Chiara De Leonibus, Carmine Settembre, Alberto Auricchio
Enzyme replacement therapy (ERT) is the standard of care for several lysosomal storage diseases (LSDs). ERT, however, requires multiple and costly administrations and has limited efficacy. We recently showed that a single high dose administration of adeno-associated viral vector serotype 8 (AAV2/8) is at least as effective as weekly ERT in a mouse model of mucopolysaccharidosis type VI (MPS VI). However, systemic administration of high doses of AAV might result in both cell-mediated immune responses and insertional mutagenesis...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27646604/the-mucus-barrier-to-inhaled-gene-therapy
#12
REVIEW
Gregg A Duncan, James Jung, Justin Hanes, Jung Soo Suk
Recent evidence suggests that the airway mucus gel layer may be impermeable to the viral and synthetic gene vectors used in past inhaled gene therapy clinical trials for diseases like cystic fibrosis. These findings support the logic that inhaled gene vectors that are incapable of penetrating the mucus barrier are unlikely to provide meaningful benefit to patients. In this review, we discuss the biochemical and biophysical features of mucus that contribute its barrier function, and how these barrier properties may be reinforced in patients with lung disease...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27633444/remodeling-of-the-extracellular-matrix-by-endothelial-cell-targeting-sirna-improves-the-epr-based-delivery-of-100%C3%A2-nm-particles
#13
Yu Sakurai, Tomoya Hada, Shoshiro Yamamoto, Akari Kato, Wataru Mizumura, Hideyoshi Harashima
A number of nano drug delivery systems have recently been developed for cancer treatment, most of which are based on the enhanced permeability and retention effect. The advantages of the enhanced permeability and retention effect can be attributed to immature vasculature. Herein we evaluated the intratumoral distribution of lipid nanoparticles when the VEGF receptor 2 on tumor endothelial cells was inhibited by liposomal siRNA. VEGF receptor 2 inhibition resulted in an increase in intratumoral distribution and therapeutic efficacy despite the maturation of the tumor vasculature...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27633443/linc00152-functions-as-a-competing-endogenous-rna-to-confer-oxaliplatin-resistance-and-holds-prognostic-values-in-colon-cancer
#14
Ben Yue, Donglan Cai, Chenchen Liu, Changyi Fang, Dongwang Yan
Long noncoding RNAs act as crucial regulators in plenty of human cancers, yet their potential roles and molecular mechanisms in chemoresistance are poorly understood. This study showed that a novel lncRNA, long intergenic noncoding RNA 152 (Linc00152 ), promoted tumor progression and conferred resistance to oxaliplatin (L-OHP)-induced apoptosis in vitro and in vivo. It antagonized chemosensitivity through acting as a competing endogenous RNA to modulate the expression of miR-193a-3p, and then erb-b2 receptor tyrosine kinase 4 (ERBB4)...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27633442/the-niemann-pick-c1-inhibitor-np3-47-enhances-gene-silencing-potency-of-lipid-nanoparticles-containing-sirna
#15
Haitang Wang, Yuen Yi C Tam, Sam Chen, Josh Zaifman, Roy van der Meel, Marco A Ciufolini, Pieter R Cullis
The therapeutic applications of lipid nanoparticle (LNP) formulations of small interfering RNA (siRNA), are hampered by inefficient delivery of encapsulated siRNA to the cytoplasm following endocytosis. Recent work has shown that up to 70% of endocytosed LNP-siRNA particles are recycled to the extracellular medium and thus cannot contribute to gene silencing. Niemann-Pick type C1 (NPC1) is a late endosomal/lysosomal membrane protein required for efficient extracellular recycling of endosomal contents. Here we assess the influence of NP3...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27633441/intracellular-delivery-of-anti-ppkc%C3%AE-thr538-via-protein-transduction-domain-mimics-for-immunomodulation
#16
E Ilker Ozay, Gabriela Gonzalez-Perez, Joe A Torres, Jyothi Vijayaraghavan, Rebecca Lawlor, Heather L Sherman, Daniel T Garrigan, Amy S Burnside, Barbara A Osborne, Gregory N Tew, Lisa M Minter
Targeting cellular proteins with antibodies, to better understand cellular signaling pathways in the context of disease modulation, is a fast-growing area of investigation. Humanized antibodies are increasingly gaining attention for their therapeutic potential, but the collection of cellular targets is limited to those secreted from cells or expressed on the cell surface. This approach leaves a wealth of intracellular proteins unexplored as putative targets for antibody binding. Protein kinase Cθ (PKCθ) is essential to T cell activation, proliferation, and differentiation, and its phosphorylation at specific residues is required for its activity...
December 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27916998/in-this-issue
#17
(no author information available yet)
No abstract text is available yet for this article.
November 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27916997/the-ema-framework-of-collaboration-with-academic-stakeholders
#18
EDITORIAL
Anton Ussi, Giovanni Migliaccio
No abstract text is available yet for this article.
November 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27916996/a-new-agent-in-the-strategy-to-cure-aids
#19
John A Zaia
No abstract text is available yet for this article.
November 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/27916995/prevention-of-muscle-wasting-by-crispr-cas9-mediated-disruption-of-myostatin-in-vivo
#20
Yuda Wei, Yanhao Chen, Yan Qiu, Huan Zhao, Gaigai Liu, Yongxian Zhang, Qingyang Meng, Guohao Wu, Yixiong Chen, Xiaolong Cai, Hui Wang, Hao Ying, Bin Zhou, Mingyao Liu, Dali Li, Qiurong Ding
No abstract text is available yet for this article.
November 2016: Molecular Therapy: the Journal of the American Society of Gene Therapy
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