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adeno-associated viral

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https://www.readbyqxmd.com/read/28105850/next-generation-tau-models-in-alzheimer-s%C3%A2-disease-research-virus-based-gene-delivery-systems
#1
V Cubinková, B Valachová, V Brezovaková, R Szabó, I Zimová, Z Kostecká, S Jadhav
Alzheimer's disease (AD) the most common form of dementia is characterized by cognitive decline and progressive loss of neurons in the central nervous system. Despite huge scientific progress, there are only few animal models that recapitulate at least majority of the AD pathology and related symptomatology. Therefore, alternative methods to develop animal models for neurodegenerative diseases are constantly explored. Recently, recombinant adeno-associated viruses (AAVs) are widely used viral vectors in development of novel models for neurodegenerative diseases...
January 19, 2017: Acta Virologica
https://www.readbyqxmd.com/read/28100454/gene-therapy-targeting-oligodendrocytes-provides-therapeutic-benefit-in-a-leukodystrophy-model
#2
Elena Georgiou, Kyriaki Sidiropoulou, Jan Richter, Christos Papaneophytou, Irene Sargiannidou, Alexia Kagiava, Georg von Jonquieres, Christina Christodoulou, Matthias Klugmann, Kleopas A Kleopa
Pelizaeus-Merzbacher-like disease or hypomyelinating leukodystrophy-2 is an autosomal recessively inherited leukodystrophy with childhood onset resulting from mutations in the gene encoding the gap junction protein connexin 47 (Cx47, encoded by GJC2). Cx47 is expressed specifically in oligodendrocytes and is crucial for gap junctional communication throughout the central nervous system. Previous studies confirmed that a cell autonomous loss-of-function mechanism underlies hypomyelinating leukodystrophy-2 and that transgenic oligodendrocyte-specific expression of another connexin, Cx32 (GJB1), can restore gap junctions in oligodendrocytes to achieve correction of the pathology in a disease model...
January 18, 2017: Brain: a Journal of Neurology
https://www.readbyqxmd.com/read/28098415/intracranial-aav-ifn-%C3%AE-gene-therapy-eliminates-invasive-xenograft-glioblastoma-and-improves-survival-in-orthotopic-syngeneic-murine-model
#3
Dwijit GuhaSarkar, James Neiswender, Qin Su, Guangping Gao, Miguel Sena-Esteves
The highly invasive property of glioblastoma (GBM) cells and genetic heterogeneity are largely responsible for tumor recurrence after the current standard-of-care treatment and thus a direct cause of death. Previously, we have shown that intracranial interferon-beta (IFN-β) gene therapy by locally administered adeno-associated viral vectors (AAV) successfully treats noninvasive orthotopic glioblastoma models. Here, we extend these findings by testing this approach in invasive human GBM xenograft and syngeneic mouse models...
November 9, 2016: Molecular Oncology
https://www.readbyqxmd.com/read/28094877/hypothalamus-specific-re-introduction-of-snord116-into-otherwise-snord116-deficient-mice-increased-energy-expenditure
#4
Y Qi, L Purtell, M Fu, L Zhang, S Zolotukhin, L Campbell, H Herzog
The Snord116 gene cluster has been recognized as a critical contributor to the Prader-Willi Syndrome (PWS) with mice lacking Snord116 displaying many classical PWS phenotypes including low postnatal body weight, reduced bone mass and increased food intake. However, these mice do not develop obesity due to increased energy expenditure. To understand the physiological function of Snord116 better and potentially rescue the altered metabolism of Snord116(-/-) mice, we used an adeno-associated viral (AAV) approach to reintroduce the Snord116 gene product into the hypothalamus in Snord116(-/-) mice at different ages...
January 17, 2017: Journal of Neuroendocrinology
https://www.readbyqxmd.com/read/28059103/radioiodinated-capsids-facilitate-in-vivo-non-invasive-tracking-of-adeno-associated-gene-transfer-vectors
#5
P Kothari, B P De, B He, A Chen, M J Chiuchiolo, D Kim, A Nikolopoulou, A Amor-Coarasa, J P Dyke, H U Voss, S M Kaminsky, C P Foley, S Vallabhajosula, B Hu, S G DiMagno, D Sondhi, R G Crystal, J W Babich, D Ballon
Viral vector mediated gene therapy has become commonplace in clinical trials for a wide range of inherited disorders. Successful gene transfer depends on a number of factors, of which tissue tropism is among the most important. To date, definitive mapping of the spatial and temporal distribution of viral vectors in vivo has generally required postmortem examination of tissue. Here we present two methods for radiolabeling adeno-associated virus (AAV), one of the most commonly used viral vectors for gene therapy trials, and demonstrate their potential usefulness in the development of surrogate markers for vector delivery during the first week after administration...
January 6, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28056565/characterization-of-aav-mediated-human-factor-viii-gene-therapy-in-hemophilia-a-mice
#6
Jenny A Greig, Qiang Wang, Amanda L Reicherter, Shu-Jen Chen, Alexandra L Hanlon, Christopher H Tipper, K Reed Clark, Samuel Wadsworth, Lili Wang, James M Wilson
Adeno-associated viral (AAV) vectors are promising vehicles for hemophilia gene therapy, with favorable clinical trial data seen in the treatment of hemophilia B. In an effort to optimize the expression of human coagulation factor VIII (hFVIII) for the treatment of hemophilia A, we performed an extensive study with numerous combinations of liver-specific promoter and enhancer elements with a codon-optimized hFVIII transgene. After generating 42 variants of three reduced-size promoters and three small enhancers, transgene cassettes were packaged within a single AAV capsid, AAVrh10, to eliminate performance differences due to the capsid type...
January 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28042944/comparison-of-serum-raav-serotype-specific-antibodies-in-patients-with-duchenne-muscular-dystrophy-becker-muscular-dystrophy-inclusion-body-myositis-or-gne-myopathy
#7
Deborah Zygmunt, Kelly E Crowe, Kevin Flanigan, Paul T Martin
Recombinant Adeno-associated virus (rAAV) is a commonly used gene therapy vector for the delivery of therapeutic transgenes in a variety of human diseases, but pre-existing serum antibodies to viral capsid proteins can greatly inhibit rAAV transduction of tissues. We have assayed serum from patients with Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (DMD), Inclusion Body Myositis (IBM), and GNE myopathy (GNE). These were compared to serum from otherwise normal human subjects to determine the extent of pre-existing serum antibodies to rAAVrh74, rAAV1, rAAV2, rAAV6, rAAV8 and rAAV9...
January 2, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28017835/aav-mediated-transfer-of-rhoa-shrna-and-cntf-promotes-retinal-ganglion-cell-survival-and-axon-regeneration
#8
Ling-Ping Cen, Jia-Jian Liang, Jian-Huan Chen, Alan R Harvey, Tsz Kin Ng, Mingzhi Zhang, Chi Pui Pang, Qi Cui, You-Ming Fan
The aim of the present study was to determine whether adeno-associated viral vector (AAV) mediated transfer of ciliary neurotrophic factor (CNTF) and RhoA shRNA has additive effects on promoting the survival and axon regeneration of retinal ganglion cells (RGCs) after optic nerve crush (ONC). Silencing effects of AAV-RhoA shRNA were confirmed by examining neurite outgrowth in PC12 cells, and by quantifying RhoA expression levels with western blotting. Young adult Fischer rats received an intravitreal injection of (i) saline, (ii) AAV green fluorescent protein (GFP), (iii) AAV-CNTF, (iv) AAV-RhoA shRNA, or (v) a combination of both AAV-CNTF and AAV-RhoA shRNA...
December 23, 2016: Neuroscience
https://www.readbyqxmd.com/read/28012171/mir-15b-mediates-oxaliplatin-induced-chronic-neuropathic-pain-through-bace1-downregulation
#9
Naomi Ito, Atsushi Sakai, Noriko Miyake, Motoyo Maruyama, Hirotoshi Iwasaki, Koichi Miyake, Takashi Okada, Atsuhiro Sakamoto, Hidenori Suzuki
BACKGROUND AND PURPOSE: Although oxaliplatin is an effective anti-cancer platinum compound, it can cause painful chronic neuropathy, and its molecular mechanisms remain poorly understood. MicroRNAs are small non-coding RNAs that negatively regulate gene expression in a sequence-specific manner. Although microRNAs have been increasingly recognised as important modulators in a variety of pain conditions, their involvement in chemotherapy-induced neuropathic pain is unknown. EXPERIMENTAL APPROACH: Oxaliplatin-induced chronic neuropathic pain was induced in rats by intraperitoneal oxaliplatin injections (2 mg kg(-1) ) for 5 consecutive days...
December 23, 2016: British Journal of Pharmacology
https://www.readbyqxmd.com/read/27999185/early-and-progressive-deficit-of-neuronal-activity-patterns-in-a-model-of-local-amyloid-pathology-in-mouse-prefrontal-cortex
#10
Fani Koukouli, Marie Rooy, Uwe Maskos
Alzheimer's Disease (AD) is the most common form of dementia. The condition predominantly affects the cerebral cortex and hippocampus and is characterized by the spread of amyloid plaques and neurofibrillary tangles (NFTs). But soluble amyloid-β (Aβ) oligomers have also been identified to accumulate in the brains of AD patients and correlate with cognitive dysfunction more than the extent of plaque deposition. Here, we developed an adeno-associated viral vector expressing the human mutated amyloid precursor protein (AAV-hAPP)...
December 20, 2016: Aging
https://www.readbyqxmd.com/read/27995572/intramuscular-delivery-of-scaav9-higf1-prolongs-survival-in-the-hsod1-g93a-als-mouse-model-via-upregulation-of-d-amino-acid-oxidase
#11
HuiQian Lin, HaoJie Hu, WeiSong Duan, YaLing Liu, GuoJun Tan, ZhongYao Li, YaKun Liu, BinBin Deng, XueQin Song, Wan Wang, Di Wen, Ying Wang, ChunYan Li
Self-complementary adeno-associated viral vector 9 (scAAV9) has been confirmed to be an efficient AAV serotype for gene transfer to the central nervous system (CNS). Neurotrophic factors have been considered to be therapeutic targets for amyotrophic lateral sclerosis (ALS). In the present study, we intramuscularly injected scAAV9 encoding human insulin-like growth factor 1 (hIGF1) into an hSOD1(G93A) ALS mouse model. We observed that scAAV9-hIGF1 significantly reduced the loss of motor neurons of the anterior horn in the lumbar spinal cord and delayed muscle atrophy in ALS mice...
December 19, 2016: Molecular Neurobiology
https://www.readbyqxmd.com/read/27990448/transcriptional-activity-of-novel-aldh1l1-promoters-in-the-rat-brain-following-aav-vector-mediated-gene-transfer
#12
Janitha M Mudannayake, Alexandre Mouravlev, Dahna M Fong, Deborah Young
Aldehyde dehydrogenase family 1, member L1 (ALDH1L1) is a recently characterized pan-astrocytic marker that is more homogenously expressed throughout the brain than the classic astrocytic marker, glial fibrillary acidic protein. We generated putative promoter sequence variants of the rat ALDH1L1 gene for use in adeno-associated viral vector-mediated gene transfer, with an aim to achieve selective regulation of transgene expression in astrocytes in the rat brain. Unexpectedly, ALDH1L1 promoter variants mediated transcriptional activity exclusively in neurons in the substantia nigra pars compacta as assessed by luciferase reporter expression at 3 weeks postvector infusion...
2016: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/27989734/systemic-application-of-aav-vectors-targeting-gfap-expressing-astrocytes-in-z-q175-ki-huntington-s-disease-mice
#13
Tatyana Vagner, Anton Dvorzhak, Anna Maria Wójtowicz, Christoph Harms, Rosemarie Grantyn
Huntington's disease (HD) affects both neurons and astrocytes. To target the latter and to ensure brain-wide transgene expression, adeno-associated viral (AAV) vectors can be administered intravenously, as AAV vectors cross the blood-brain barrier (BBB) and enable preferential transduction of astrocytes due to their close association with blood vessels. However, there is a possibility that the subclass of GFAP-expressing astrocytes performs a distinct role in HD and reacts differently to therapeutic measures than the rest of the astrocytes...
October 27, 2016: Molecular and Cellular Neurosciences
https://www.readbyqxmd.com/read/27989459/aav-mediated-anterograde-transsynaptic-tagging-mapping-corticocollicular-input-defined-neural-pathways-for-defense-behaviors
#14
Brian Zingg, Xiao-Lin Chou, Zheng-Gang Zhang, Lukas Mesik, Feixue Liang, Huizhong Whit Tao, Li I Zhang
To decipher neural circuits underlying brain functions, viral tracers are widely applied to map input and output connectivity of neuronal populations. Despite the successful application of retrograde transsynaptic viruses for identifying presynaptic neurons of transduced neurons, analogous anterograde transsynaptic tools for tagging postsynaptically targeted neurons remain under development. Here, we discovered that adeno-associated viruses (AAV1 and AAV9) exhibit anterograde transsynaptic spread properties...
January 4, 2017: Neuron
https://www.readbyqxmd.com/read/27978950/gene-therapy-with-angiotensin-1-9-preserves-left-ventricular-systolic-function-after-myocardial-infarction
#15
Caroline Fattah, Katrin Nather, Charlotte S McCarroll, Maria P Hortigon-Vinagre, Victor Zamora, Monica Flores-Munoz, Lisa McArthur, Lorena Zentilin, Mauro Giacca, Rhian M Touyz, Godfrey L Smith, Christopher M Loughrey, Stuart A Nicklin
BACKGROUND: Angiotensin-(1-9) [Ang-(1-9)] is a novel peptide of the counter-regulatory axis of the renin-angiotensin-aldosterone system previously demonstrated to have therapeutic potential in hypertensive cardiomyopathy when administered via osmotic mini-pump. Here, we investigate whether gene transfer of Ang-(1-9) is cardioprotective in a murine model of myocardial infarction (MI). OBJECTIVES: The authors evaluated effects of Ang-(1-9) gene therapy on myocardial structural and functional remodeling post-infarction...
December 20, 2016: Journal of the American College of Cardiology
https://www.readbyqxmd.com/read/27940927/adeno-associated-virus-mediated-gene-transfer-into-taste-cells-in-vivo
#16
Akiyuki Taruno, Makiko Kashio, Hongxin Sun, Kenta Kobayashi, Hiromi Sano, Atsushi Nambu, Yoshinori Marunaka
The sense of taste is achieved by cooperation of many signaling molecules expressed in taste cells, which code and transmit information on quality and intensity of taste to the nervous system. Viral vector-mediated gene transfer techniques have been proven to be useful to study and control function of a gene product in vivo However, there is no transduction method for taste cells in live animals. Here, we have established a method for inducing foreign gene expression in mouse taste cells in vivo by recombinant adeno-associated virus (AAV) vector...
January 2017: Chemical Senses
https://www.readbyqxmd.com/read/27939396/a-role-for-peroxisome-proliferator-activated-receptor-gamma-coactivator-1%C3%AE-in-nucleus-accumbens-neuron-subtypes-in-cocaine-action
#17
Ramesh Chandra, Michel Engeln, T Chase Francis, Prasad Konkalmatt, Dipal Patel, Mary Kay Lobo
BACKGROUND: Molecules critically involved in cocaine behavioral plasticity are known to regulate and interact with peroxisome proliferator-activated receptor gamma coactivator-1α (PGC-1α). In addition, the PGC-1α promoter has binding sites for early growth response 3 (Egr3), which plays a dynamic role in cocaine action in nucleus accumbens (NAc) medium spiny neuron (MSN) subtypes, those enriched in dopamine receptor D1 (D1-MSN) versus D2 (D2-MSN). However, the role of PGC-1α in NAc in cocaine action is unknown...
October 28, 2016: Biological Psychiatry
https://www.readbyqxmd.com/read/27937051/wnt10b-and-dkk-1-gene-therapy-differentially-influenced-trabecular-bone-architecture-soft-tissue-integrity-and-osteophytosis-in-a-skeletally-mature-rat-model-of-osteoarthritis
#18
Jeffrey B Mason, Brittney L Gurda, Kurt D Hankenson, Lindsey R Harper, Cathy S Carlson, James M Wilson, Dean W Richardson
AIMS: Our goals in the current experiments were to determine if 1) up-regulation of Wnt signaling would induce OA changes in stable stifle joints and 2) if down-regulation of Wnt signaling in destabilized joints would influence the progression of OA. METHODS: At 37 weeks of age, rats were injected in the stifle joint with a recombinant adeno-associated viral vector containing the Wnt-inhibitor Dkk-1 or a Wnt10b transgene. At 40 weeks of age, rats underwent surgical destabilization of the joint...
December 9, 2016: Connective Tissue Research
https://www.readbyqxmd.com/read/27934080/remote-control-of-light-triggered-virotherapy
#19
S-Ja Tseng, Kuo-Yen Huang, Ivan M Kempson, Shih-Han Kao, Meng-Chia Liu, Shuenn-Chen Yang, Zi-Xian Liao, Pan-Chyr Yang
Clinical virotherapy has been successfully approved for use in cancer treatment by the U.S. Food and Drug Administration; however, a number of improvements are still sought to more broadly develop virotherapy. A particular challenge is to administer viral therapy systemically and overcome limitations in intratumoral injection, especially for complex tumors within sensitive organs. To achieve this, however, a technique is required that delivers the virus to the tumor before the body's natural self-defense eradicates the virus prematurely...
November 22, 2016: ACS Nano
https://www.readbyqxmd.com/read/27933310/dynamics-of-antigen-presentation-to-transgene-product-specific-cd4-t-cells-and-of-treg-induction-upon-hepatic-aav-gene-transfer
#20
George Q Perrin, Irene Zolotukhin, Alexandra Sherman, Moanaro Biswas, Ype P de Jong, Cox Terhorst, Andrew M Davidoff, Roland W Herzog
The tolerogenic hepatic microenvironment impedes clearance of viral infections but is an advantage in viral vector gene transfer, which often results in immune tolerance induction to transgene products. Although the underlying tolerance mechanism has been extensively studied, our understanding of antigen presentation to transgene product-specific CD4(+) T cells remains limited. To address this, we administered hepatotropic adeno-associated virus (AAV8) vector expressing cytoplasmic ovalbumin (OVA) into wt mice followed by adoptive transfer of transgenic OVA-specific T cells...
2016: Molecular Therapy. Methods & Clinical Development
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