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https://www.readbyqxmd.com/read/29149775/lentiviral-vectors-and-adeno-associated-virus-vectors-useful-tools-for-gene-transfer-in-pain-research
#1
REVIEW
Zheng Chen-Xi, Wang Sheng-Ming, Bai Yun-Hu, Luo Ting-Ting, Wang Jia-Qi, Dai Chun-Qiu, Guo Bao-Lin, Luo Shi-Cheng, Wang Dong-Hui, Yang Yan-Ling, Wang Ya-Yun
Pain, especially chronic pain, has always been a heated point in both basic and clinical researches since it puts heavy burdens on both individuals and the whole society. A better understanding of the role of biological molecules and various ionic channels involved in pain can shed light on the mechanism under pain and advocate the development of pain management. Using viral vectors to transfer specific genes at targeted sites is a promising method for both research and clinical applications. Lentiviral vectors and adeno-associated virus (AAV) vectors which allow stable and long-term expression of transgene in non-dividing cells are widely applied in pain research...
November 17, 2017: Anatomical Record: Advances in Integrative Anatomy and Evolutionary Biology
https://www.readbyqxmd.com/read/29146410/cart-neurons-in-the-arcuate-nucleus-and-lateral-hypothalamic-area-exert-differential-controls-on-energy-homeostasis
#2
Jackie Lau, Aitak Farzi, Yue Qi, Regine Heilbronn, Mario Mietzsch, Yan-Chuan Shi, Herbert Herzog
OBJECTIVE: The cocaine- and amphetamine-regulated transcript (CART) codes for a pivotal neuropeptide important in the control of appetite and energy homeostasis. However, limited understanding exists for the defined effector sites underlying CART function, as discrepant effects of central CART administration have been reported. METHODS: By combining Cart-cre knock-in mice with a Cart adeno-associated viral vector designed using the flip-excision switch (AAV-FLEX) technology, specific reintroduction or overexpression of CART selectively in CART neurons in the arcuate nucleus (Arc) and lateral hypothalamic area (LHA), respectively, was achieved...
November 8, 2017: Molecular Metabolism
https://www.readbyqxmd.com/read/29130354/delivery-of-adeno-associated-viral-vectors-in-adult-mammalian-inner-ear-cell-subtypes-without-auditory-dysfunction
#3
Yong Tao, Mingqian Huang, Yilai Shu, Adam Ruprecht, Hongyang Wang, Yong Tang, Luk H Vandenberghe, Qiuju Wang, Guangping Gao, Wei-Jia Kong, Zheng-Yi Chen
Hearing loss, including genetic hearing loss, is one of the most common forms of sensory deficits in human with limited options of treatment. Adeno-associated virus (AAV)-mediated gene transfer has been shown to effectively recover auditory functions in mouse models of genetic deafness when delivered at neonatal stages. However, the mouse cochlea is still developing at those time points whereas in human the newborn inner ears are already fully mature. For effective gene therapy to treat genetic deafness, it is necessary to determine whether or not AAV-mediated therapy can be equally effective in the fully mature mouse inner ear without causing damages to the inner ear...
November 12, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29130152/combining-engineered-nucleases-with-adeno-associated-viral-vectors-for-therapeutic-gene-editing
#4
Benjamin E Epstein, David V Schaffer
With the recent advent of several generations of targeted DNA nucleases, most recently CRISPR/Cas9, genome editing has become broadly accessible across the biomedical community. Importantly, the capacity of these nucleases to modify specific genomic loci associated with human disease could render new classes of genetic disease, including autosomal dominant or even idiopathic disease, accessible to gene therapy. In parallel, the emergence of adeno-associated virus (AAV) as a clinically important vector raises the possibility of integrating these two technologies towards the development of gene editing therapies...
2017: Advances in Experimental Medicine and Biology
https://www.readbyqxmd.com/read/29127625/gene-therapy-for-hemophilia-progress-to-date
#5
John C Chapin, Paul Edward Monahan
Hemophilia is a congenital bleeding disorder that affects nearly half a million individuals worldwide. Joint bleeding and other co-morbidities are a significant source of debilitation for this population. Current therapies are effective but must be given lifelong at regular intervals, are costly, and are available to only about 25% of the hemophilia population living in resource-rich countries. Gene therapy for hemophilia has been in development for three decades and is now entering pivotal-stage clinical trials...
November 10, 2017: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
https://www.readbyqxmd.com/read/29120948/parvalbumin-containing-gaba-cells-and-schizophrenia-experimental-model-based-on-targeted-gene-delivery-through-adeno-associated-viruses
#6
Marta U Woloszynowska-Fraser, Peer Wulff, Gernot Riedel
Understanding the contribution of transmitter systems in behavioural pharmacology has a long tradition. Multiple techniques such as transmitter-specific lesions, and also localized administration of pharmacological toxins including agonists and antagonists of selected receptors have been applied. More recently, modern genetic tools have permitted cell-type selective interferences, for example by expression of light-sensitive channels followed by optogenetic stimulation in behaviourally meaningful settings or by engineered channels termed DREADDS that respond to peripherally administered drugs...
December 2017: Behavioural Pharmacology
https://www.readbyqxmd.com/read/29117317/superior-retinal-gene-transfer-and-biodistribution-profile-of-subretinal-versus-intravitreal-delivery-of-aav8-in-nonhuman-primates
#7
Immanuel P Seitz, Stylianos Michalakis, Barbara Wilhelm, Felix F Reichel, G Alex Ochakovski, Eberhart Zrenner, Marius Ueffing, Martin Biel, Bernd Wissinger, Karl U Bartz-Schmidt, Tobias Peters, M Dominik Fischer
Purpose: To investigate shedding and biodistribution characteristics of recombinant adeno-associated virus serotype 8 (rAAV8) after single-dose subretinal or intravitreal injection in nonhuman primates (NHP, Macaca fascicularis) as a surrogate for environmental hazard and patient safety. Methods: In a study for regulatory submission, 22 NHP were divided into four cohorts receiving either single subretinal injections of vehicle or clinical grade rAAV8 (1 × 1011 or 1 × 1012 vector genomes [vg]) versus single intravitreal application of 1 × 1012 vg...
November 1, 2017: Investigative Ophthalmology & Visual Science
https://www.readbyqxmd.com/read/29116194/site-specific-modification-of-adeno-associated-virus-enables-both-fluorescent-imaging-of-viral-particles-and-characterization-of-the-capsid-interactome
#8
Jayanth S Chandran, Paul S Sharp, Evangelia Karyka, João Miguel da Conceição Aves-Cruzeiro, Ian Coldicott, Lydia Castelli, Guillaume Hautbergue, Mark O Collins, Mimoun Azzouz
Adeno-associated viruses (AAVs) are attractive gene therapy vectors due to their low toxicity, high stability, and rare integration into the host genome. Expressing ligands on the viral capsid can re-target AAVs to new cell types, but limited sites have been identified on the capsid that tolerate a peptide insertion. Here, we incorporated a site-specific tetracysteine sequence into the AAV serotype 9 (AAV9) capsid, to permit labelling of viral particles with either a fluorescent dye or biotin. We demonstrate that fluorescently labelled particles are detectable in vitro, and explore the utility of the method in vivo in mice with time-lapse imaging...
November 7, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29109058/homer2-within-the-central-nucleus-of-the-amygdala-modulates-withdrawal-induced-anxiety-in-a-mouse-model-of-binge-drinking
#9
K M Lee, M A Coelho, K R Sern, K K Szumlinski
A history of binge-drinking decreases protein expression of the glutamate-related scaffolding protein Homer2 within the central nucleus of the amygdala (CEA), coinciding with behavioral signs of negative affect. To assess the functional relevance of this protein change for withdrawal-induced hyper-anxiety, adult (PND 56) and adolescent (PND 28) male C57BL/6J mice were administered an intra-CEA infusion of an adeno-associated viral vector (AAV) carrying either cDNA to express Homer2 (H2-cDNA) or GFP as control...
November 3, 2017: Neuropharmacology
https://www.readbyqxmd.com/read/29106033/brain-5-lipoxygenase-over-expression-worsens-memory-synaptic-integrity-and-tau-pathology-in-the-p301s-mice
#10
Alana N Vagnozzi, Phillip F Giannopoulos, Domenico Praticò
Progressive accumulation of highly phosphorylated tau protein isoforms is the main feature of a group of neurodegenerative diseases collectively called tauopathies. Data from human and animal models of these diseases have shown that neuroinflammation often accompanies their pathogenesis. The 5-lipoxygenase (5LO) is an enzyme widely expressed in the brain and a source of potent pro-inflammatory mediators, while its pharmacological inhibition modulates the phenotype of a tau transgenic mouse model, the htau mice...
November 4, 2017: Aging Cell
https://www.readbyqxmd.com/read/29101166/transcription-factor-%C3%AE-fosb-acts-within-the-nucleus-of-the-solitary-tract-to-increase-mean-arterial-pressure-during-exposures-to-intermittent-hypoxia
#11
Qiong Wu, J Thomas Cunningham, Steve W Mifflin
ΔFosB is a member of the AP-1 family of transcription factors. ΔFosB has low constitutive expression in the central nervous system and is induced following exposure of rodents to intermittent hypoxia (IH), a model of the arterial hypoxemia that accompanies sleep apnea. We hypothesize ΔFosB in the nucleus of solitary tract (NTS) contributes to increased mean arterial pressure (MAP) during IH. The NTS of 11 male Sprague Dawley rats was injected (3 sites, 100nl per site) with a dominant-negative against ΔFosB (ΔJunD) in an adeno-associated viral vector (AAV)-green fluorescent protein (GFP) reporter...
November 3, 2017: American Journal of Physiology. Heart and Circulatory Physiology
https://www.readbyqxmd.com/read/29099403/considerations-for-the-use-of-virally-delivered-genetic-tools-for-in-vivo-circuit-analysis-and-behavior-in-mutant-mice-a-practical-guide-to-optogenetics
#12
Dóra Zelena, Kornél Demeter, József Haller, Diána Balázsfi
Optogenetics was the method of the year in 2010 according to Nature Neuroscience. Since then, this method has become widespread, the use of virally delivered genetic tools has extended to other fields such as pharmacogenetics, and optogenetic techniques have become frequently applied in genetically manipulated animals for in-vivo circuit analysis and behavioral studies. However, several issues should be taken into consideration when planning such experiments. We aimed to summarize the critical points concerning optogenetic manipulation of a specific brain area in mutant mice...
December 2017: Behavioural Pharmacology
https://www.readbyqxmd.com/read/29091557/single-dose-gene-replacement-therapy-for-spinal-muscular-atrophy
#13
Jerry R Mendell, Samiah Al-Zaidy, Richard Shell, W Dave Arnold, Louise R Rodino-Klapac, Thomas W Prior, Linda Lowes, Lindsay Alfano, Katherine Berry, Kathleen Church, John T Kissel, Sukumar Nagendran, James L'Italien, Douglas M Sproule, Courtney Wells, Jessica A Cardenas, Marjet D Heitzer, Allan Kaspar, Sarah Corcoran, Lyndsey Braun, Shibi Likhite, Carlos Miranda, Kathrin Meyer, K D Foust, Arthur H M Burghes, Brian K Kaspar
BACKGROUND: Spinal muscular atrophy type 1 (SMA1) is a progressive, monogenic motor neuron disease with an onset during infancy that results in failure to achieve motor milestones and in death or the need for mechanical ventilation by 2 years of age. We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease. METHODS: Fifteen patients with SMA1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complementary DNA encoding the missing SMN protein...
November 2, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29070854/altered-norbin-expression-in-patients-with-epilepsy-and-a-rat-model
#14
Yali Xu, Zengyou Li, Li Yao, Xingping Zhang, Dan Gan, Manchun Jiang, Na Wang, Guojun Chen, Xuefeng Wang
Norbin is widely distributed in neuronal tissues, is a regulator of Ca2(+)/calmodulin-dependent protein kinase II (CaMKII) phosphorylation. Norbin is also an important endogenous modulator of metabotropic glutamate receptor 5 (mGluR5) signaling, and nervous system-specific homozygous gene disruptions, result in epileptic seizures. In this study, we aimed to investigate norbin expression patterns in epilepsy and to elucidate the relationships between norbin and mGluR5 and p-CaMKII in epilepsy. Double-immunolabeling, immunohistochemistry and immunoblotting studies showed that norbin was downregulated in the temporal neocortex of patients with temporal lobe epilepsy (TLE) compared with control subjects...
October 25, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29069788/the-sirtuin-6-prevents-angiotensin-ii-mediated-myocardial-fibrosis-and-injury-by-targeting-ampk-ace2-signaling
#15
Zhen-Zhou Zhang, Yu-Wen Cheng, Hai-Yan Jin, Qing Chang, Qian-Hui Shang, Ying-Le Xu, Lin-Xi Chen, Ran Xu, Bei Song, Jiu-Chang Zhong
Sirtuin 6 (SIRT6) is an important modulator of cardiovascular functions in health and diseases. However, the exact role of SIRT6 in heart disease is poorly defined. We hypothesized that SIRT6 is a negative regulator of angiotensin II (Ang II)-mediated myocardial remodeling, fibrosis and injury. The male Sprague-Dawley rats were randomized to Ang II (200 ng/kg/min) infusion with an osmotic minipump and pretreated with recombinant plasmids adeno-associated viral vector (AAV)-SIRT6 (pAAV-SIRT6) or pAAV-GFP for 4 weeks...
September 22, 2017: Oncotarget
https://www.readbyqxmd.com/read/29050992/the-impact-of-13-valent-pneumococcal-conjugate-vaccination-on-virus-associated-community-acquired-pneumonia-in-elderly
#16
Susanne M Huijts, Frank E J Coenjaerts, Marieke Bolkenbaas, Cornelis H van Werkhoven, Diederick E Grobbee, Marc J M Bonten
OBJECTIVES: Our objective was to evaluate whether vaccination with the 13-valent pneumococcal conjugate vaccine (PCV13) prevents the incidence of community-acquired pneumonia (CAP) caused by influenza (Influenza-Associated CAP, IA-CAP) or other respiratory viruses in elderly. METHODS: This analysis was part of the Community-Acquired Pneumonia immunization Trial in Adults (CAPiTA); a double blind, randomized, placebo-controlled trial in 84,496 immunocompetent individuals aged ≥65 years...
October 16, 2017: Clinical Microbiology and Infection
https://www.readbyqxmd.com/read/29048971/protocol-for-efficient-generation-and-characterization-of-adeno-associated-viral-vectors
#17
Andreas Jungmann, Barbara Leuchs, Jean Rommelaere, Hugo A Katus, Oliver J Müller
Adeno-associated virus vectors are a powerful tool for gene transfer approaches. We have established a simple and fast plasmid-based production system for achieving high adeno-associated virus titers within 6 working days. The same procedure can be used for all serotypes and thus allows direct comparability of different serotypes. In this protocol we describe a step-by-step procedure that results in well-characterized vectors suitable for both in vitro approaches and preclinical studies.
October 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/29042259/widespread-optogenetic-expression-in-macaque-cortex-obtained-with-mr-guided-convection-enhanced-delivery-ced-of-aav-vector-to-the-thalamus
#18
Azadeh Yazdan-Shahmorad, Nan Tian, Viktor Kharazia, Lluis Samaranch, Adrian Kells, John Bringas, Jiwei He, Krystof Bankiewicz, Philip N Sabes
BACKGROUND: In non-human primate (NHP) optogenetics, infecting large cortical areas with viral vectors is often a difficult and time-consuming task. Previous work has shown that parenchymal delivery of adeno-associated virus (AAV) in the thalamus by convection-enhanced delivery (CED) can lead to large-scale transduction via axonal transport in distal areas including cortex. We used this approach to obtain widespread cortical expression of light-sensitive ion channels. NEW METHOD: AAV vectors co-expressing channelrhodopsin-2 (ChR2) and yellow fluorescent protein (YFP) genes were infused into thalamus of three rhesus macaques under MR-guided CED...
October 16, 2017: Journal of Neuroscience Methods
https://www.readbyqxmd.com/read/29018836/one-step-fabrication-of-bone-morphogenetic-protein-2-gene-activated-porous-poly-l-lactide-scaffold-for-bone-induction
#19
Jingwen Xue, Hang Lin, Allison Bean, Ying Tang, Jian Tan, Rocky S Tuan, Bing Wang
Bone morphogenetic protein 2 (BMP2) is an efficacious inducer for the osteogenesis of mesenchymal stem cells (MSCs). Conventional applications of BMP2 have involved either the direct incorporation of BMP2 protein or ex vivo BMP2 gene transfer into stem cells prior to their transplantation. These approaches are able to promote bone formation to some extent; however, they are hampered by either the lack of stability and sustainability of BMP2 protein or the time-consuming and cost-prohibitive in vitro cell culture procedure...
December 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29016640/effects-of-aav-mediated-knockdown-of-nnos-and-gpx-1-gene-expression-in-rat-hippocampus-after-traumatic-brain-injury
#20
Deborah R Boone, Jeanna M Leek, Michael T Falduto, Karen E O Torres, Stacy L Sell, Margaret A Parsley, Jeremy C Cowart, Tatsuo Uchida, Maria-Adelaide Micci, Douglas S DeWitt, Donald S Prough, Helen L Hellmich
Virally mediated RNA interference (RNAi) to knock down injury-induced genes could improve functional outcome after traumatic brain injury (TBI); however, little is known about the consequences of gene knockdown on downstream cell signaling pathways and how RNAi influences neurodegeneration and behavior. Here, we assessed the effects of adeno-associated virus (AAV) siRNA vectors that target two genes with opposing roles in TBI pathogenesis: the allegedly detrimental neuronal nitric oxide synthase (nNOS) and the potentially protective glutathione peroxidase 1 (GPx-1)...
2017: PloS One
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