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adeno-associated

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https://www.readbyqxmd.com/read/29051551/reducing-sarcolipin-expression-mitigates-duchenne-muscular-dystrophy-and-associated-cardiomyopathy-in-mice
#1
Antanina Voit, Vishwendra Patel, Ronald Pachon, Vikas Shah, Mohammad Bakhutma, Erik Kohlbrenner, Joseph J McArdle, Louis J Dell'Italia, Jerry R Mendell, Lai-Hua Xie, Roger J Hajjar, Dongsheng Duan, Diego Fraidenraich, Gopal J Babu
Sarcolipin (SLN) is an inhibitor of the sarco/endoplasmic reticulum (SR) Ca(2+) ATPase (SERCA) and is abnormally elevated in the muscle of Duchenne muscular dystrophy (DMD) patients and animal models. Here we show that reducing SLN levels ameliorates dystrophic pathology in the severe dystrophin/utrophin double mutant (mdx:utr (-/-)) mouse model of DMD. Germline inactivation of one allele of the SLN gene normalizes SLN expression, restores SERCA function, mitigates skeletal muscle and cardiac pathology, improves muscle regeneration, and extends the lifespan...
October 20, 2017: Nature Communications
https://www.readbyqxmd.com/read/29050429/isolation-and-genetic-characterization-of-a-novel-adeno-associated-virus-from-muscovy-ducks-in-china
#2
X N Su, J J Liu, Q F Zhou, X H Zhang, L C Zhao, Q M Xie, W G Chen, F Chen
Adeno-associated virus (AAV; genus Dependoparvovirus, family Parvoviridae) was first discovered in 1965 as a contaminant in adenovirus preparations. The AAVs are generally considered non-pathogenic, and they have the ability to attenuate the replication of other more pathogenic viruses, which makes them attractive as potential therapeutics or preventative measures. This study characterized a novel AAV isolated from Muscovy ducks in China. The novel virus (MHH-05-2015) was isolated after propagating a field isolate of the DAdV-3 virus (a type 3 duck adenovirus) in duck embryo fibroblasts...
September 1, 2017: Poultry Science
https://www.readbyqxmd.com/read/29048971/protocol-for-efficient-generation-and-characterization-of-adeno-associated-viral-vectors
#3
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/29046435/long-acting-mic-1-gdf15-molecules-to-treat-obesity-evidence-from-mice-to-monkeys
#4
Yumei Xiong, Kenneth Walker, Xiaoshan Min, Clarence Hale, Thanhvien Tran, Renee Komorowski, Jerry Yang, Jasmine Davda, Noi Nuanmanee, Dao Kemp, Xiaozhen Wang, Hantao Liu, Silke Miller, Ki Jeong Lee, Zhulun Wang, Murielle M Véniant
In search of metabolically regulated secreted proteins, we conducted a microarray study comparing gene expression in major metabolic tissues of fed and fasted ob/ob mice and C57BL/6 mice. The array used in this study included probes for ~4000 genes annotated as potential secreted proteins. Circulating macrophage inhibitory cytokine 1 (MIC-1)/growth differentiation factor 15 (GDF15) concentrations were increased in obese mice, rats, and humans in comparison to age-matched lean controls. Adeno-associated virus-mediated overexpression of GDF15 and recombinant GDF15 treatments reduced food intake and body weight and improved metabolic profiles in various metabolic disease models in mice, rats, and obese cynomolgus monkeys...
October 18, 2017: Science Translational Medicine
https://www.readbyqxmd.com/read/29042811/optical-inactivation-of-the-anterior-cingulate-cortex-modulate-descending-pain-pathway-in-a-rat-model-of-trigeminal-neuropathic-pain-created-via-chronic-constriction-injury-of-the-infraorbital-nerve
#5
Hyeong Cheol Moon, Won Ik Heo, Yon Ji Kim, Daae Lee, So Yoon Won, Hong Rae Kim, Seung Man Ha, Youn Joo Lee, Young Seok Park
PURPOSE: The anterior cingulate cortex (ACC) plays a critical role in the initiation, development, and maintenance of neuropathic pain. Recently, the effects of optical stimulation on pain have been investigated, but the therapeutic effects of optical stimulation on trigeminal neuralgia (TN) have not been clearly shown. Here, we investigated the effects of optical inhibition of the ACC on TN lesions to determine whether the alleviation of pain affects behavior performance and thalamic neuron signaling...
2017: Journal of Pain Research
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
#6
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 14, 2017: Journal of Neuroscience Methods
https://www.readbyqxmd.com/read/29037791/effect-of-optogenetic-modulation-on-entopeduncular-input-affects-thalamic-discharge-and-behavior-in-an-aav2-%C3%AE-synuclein-induced-hemiparkinson-rat-model
#7
Hyeong Cheol Moon, So Yoon Won, Eung Gook Kim, Hyong Kyu Kim, Chul Bum Cho, Young Seok Park
OBJECTIVE: Neuromodulation of the globus pallidus internus(GPi) alleviates Parkinson's disease symptoms. The primate GPi is homologous to the rat entopeduncular nucleus (EP). The aim of the present study was to determine if optogenetic modulation of the EP could alter parkinsonian behavior or thalamic discharge in a hemiparkinson rat model. METHODS: We injected an adeno-associated virus type-2 expressing α-synuclein (AAV2-α-syn) into the substantia nigra pars compacta (SNc) of the right hemisphere and confirmed parkinsonian behavior using an amphetamine-induced rotation test...
October 14, 2017: Neuroscience Letters
https://www.readbyqxmd.com/read/29033566/raav-mediated-overexpression-of-tgf-%C3%AE-via-vector-delivery-in-polymeric-micelles-stimulates-the-biological-and-reparative-activities-of-human-articular-chondrocytes-in-vitro-and-in-a-human-osteochondral-defect-model
#8
Ana Rey-Rico, Jagadeesh K Venkatesan, Gertrud Schmitt, Angel Concheiro, Henning Madry, Carmen Alvarez-Lorenzo, Magali Cucchiarini
Recombinant adeno-associated virus (rAAV) vectors are clinically adapted vectors to durably treat human osteoarthritis (OA). Controlled delivery of rAAV vectors via polymeric micelles was reported to enhance the temporal and spatial presentation of the vectors into their targets. Here, we tested the feasibility of delivering rAAV vectors via poly (ethylene oxide) (PEO) and poly (propylene oxide) (PPO) (poloxamer and poloxamine) polymeric micelles as a means to overexpress the therapeutic factor transforming growth factor-beta (TGF-β) in human OA chondrocytes and in experimental human osteochondral defects...
2017: International Journal of Nanomedicine
https://www.readbyqxmd.com/read/29033008/safety-and-long-term-efficacy-of-aav4-gene-therapy-in-patients-with-rpe65-leber-congenital-amaurosis
#9
Guylène Le Meur, Pierre Lebranchu, Fanny Billaud, Oumeya Adjali, Sébastien Schmitt, Stéphane Bézieau, Yann Péréon, Romain Valabregue, Catherine Ivan, Christophe Darmon, Philippe Moullier, Fabienne Rolling, Michel Weber
The aim of this study was the evaluation of the safety and efficacy of unilateral subretinal injection of the adeno-associated vector (AAV) serotypes 2 and 4 (AAV2/4) RPE65-RPE65 vector in patients with Leber congenital amaurosis (LCA) associated with RPE65 gene deficiency. We evaluated ocular and general tolerance and visual function up to 1 year after vector administration in the most severely affected eye in nine patients with retinal degeneration associated with mutations in the RPE65 gene. Patients received either low (1...
September 19, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29032169/survival-advantage-of-both-human-hepatocyte-xenografts-and-genome-edited-hepatocytes-for-treatment-of-%C3%AE-1-antitrypsin-deficiency
#10
Florie Borel, Qiushi Tang, Gwladys Gernoux, Cynthia Greer, Ziqiong Wang, Adi Barzel, Mark A Kay, Leonard D Shultz, Dale L Greiner, Terence R Flotte, Michael A Brehm, Christian Mueller
Hepatocytes represent an important target for gene therapy and editing of single-gene disorders. In α-1 antitrypsin (AAT) deficiency, one missense mutation results in impaired secretion of AAT. In most patients, lung damage occurs due to a lack of AAT-mediated protection of lung elastin from neutrophil elastase. In some patients, accumulation of misfolded PiZ mutant AAT protein triggers hepatocyte injury, leading to inflammation and cirrhosis. We hypothesized that correcting the Z mutant defect in hepatocytes would confer a selective advantage for repopulation of hepatocytes within an intact liver...
September 25, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/29031808/specific-disruption-of-contextual-memory-recall-by-sparse-additional-activity-in-the-dentate-gyrus
#11
Hye-Yeon Cho, Mujun Kim, Jin-Hee Han
The dentate gyrus (DG) of the hippocampus is essential for contextual and spatial memory processing. While lesion or silencing of the DG impairs contextual memory encoding and recall, overly activated DG also prevents proper memory retrieval. Abnormally elevated activity in the DG is repeatedly reported in amnesic mild cognitive impairment (aMCI) patients or aged adults. Although the correlation between memory failure and abnormally active hippocampus is clear, their causal relationship or the underlying nature of such interfering activity is not well understood...
October 12, 2017: Neurobiology of Learning and Memory
https://www.readbyqxmd.com/read/29026117/involvement-of-the-accumbal-osteopontin-interacting-transmembrane-protein-168-in-methamphetamine-induced-place-preference-and-hyperlocomotion-in-mice
#12
Kequan Fu, Yoshiaki Miyamoto, Kazuya Otake, Kazuyuki Sumi, Eriko Saika, Shohei Matsumura, Naoki Sato, Yuka Ueno, Seunghee Seo, Kyosuke Uno, Shin-Ichi Muramatsu, Atsumi Nitta
Chronic exposure to methamphetamine causes adaptive changes in brain, which underlie dependence symptoms. We have found that the transmembrane protein 168 (TMEM168) is overexpressed in the nucleus accumbens of mice upon repeated methamphetamine administration. Here, we firstly demonstrate the inhibitory effect of TMEM168 on methamphetamine-induced behavioral changes in mice, and attempt to elucidate the mechanism of this inhibition. We overexpressed TMEM168 in the nucleus accumbens of mice by using an adeno-associated virus vector (NAc-TMEM mice)...
October 12, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29025729/live-cell-imaging-of-cytosolic-nadh-nad-ratio-in-hepatocytes-and-liver-slices
#13
Ricard Masia, William J McCarty, Carolina Lahmann, Jay Luther, Raymond T Chung, Martin L Yarmush, Gary Yellen
Fatty liver disease (FLD), the most common chronic liver disease in the US, may be caused by alcohol or the metabolic syndrome. Alcohol is oxidized in the cytosol of hepatocytes by alcohol dehydrogenase (ADH), which generates NADH and increases cytosolic NADH/NAD(+) ratio. The increased ratio may be important for development of FLD, but our ability to examine this question is hindered by methodological limitations. To address this, we used the genetically-encoded fluorescent sensor Peredox to obtain dynamic, real-time measurements of cytosolic NADH/NAD(+) ratio in living hepatocytes...
October 12, 2017: American Journal of Physiology. Gastrointestinal and Liver Physiology
https://www.readbyqxmd.com/read/29024785/slow-intrathecal-injection-of-raavrh10-enhances-its-transduction-of-spinal-cord-and-therapeutic-efficacy-in-a-mutant-sod1-model-of-als
#14
Dongxiao Li, Chong Liu, Chunxing Yang, Dan Wang, Dongxia Wu, Yinkuang Qi, Qin Su, Guangping Gao, Zuoshang Xu, Yansu Guo
Mutant SOD1 causes amyotrophic lateral sclerosis (ALS) by a dominant gain of toxicity. Previous studies have demonstrated therapeutic potential of mutant SOD1-RNAi delivered by intrathecal (IT) injection of recombinant adeno-associated virus (rAAV). However, optimization of delivery is needed to overcome the high degree of variation in the transduction efficiency and therapeutic efficacy. Here, on the basis of our previously defined, efficient IT injection method, we investigated the influence of injection speed on transduction efficiency in the central nervous system (CNS)...
October 9, 2017: Neuroscience
https://www.readbyqxmd.com/read/29023813/dual-catenin-loss-in-murine-liver-causes-tight-junctional-deregulation-and-progressive-intrahepatic-cholestasis
#15
Tirthadipa Pradhan-Sundd, Lili Zhou, Ravi Vats, An Jiang, Laura Molina, Sucha Singh, Minakshi Poddar, Jacquelyn M Russell, Donna B Stolz, Michael Oertel, Udayan Apte, Simon Watkins, Sarangarajan Ranganathan, Kari N Nejak-Bowen, Prithu Sundd, Satdarshan Pal Monga
β-Catenin, the downstream effector of the Wnt signaling, plays important roles in hepatic development, regeneration and tumorigenesis. However, its role at hepatocyte adherens junctions (AJ) is relatively poorly understood, chiefly due to spontaneous compensation by γ-catenin. Here, we simultaneously ablate β- and γ-catenin expression in mouse liver by interbreeding β-catenin-γ-catenin double-floxed mice and albumin-cre transgenic mice. Double knockout mice (DKO) show failure to thrive, impaired hepatocyte differentiation, cholemia, ductular reaction, progressive cholestasis, inflammation, fibrosis and tumorigenesis, which was associated with deregulation of tight junctions (TJ) and bile acid transporters, leading to early morbidity and mortality, a phenotype reminiscent of Progressive Familial Intrahepatic Cholestasis (PFIC)...
October 10, 2017: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://www.readbyqxmd.com/read/29021994/stereotaxic-adeno-associated-virus-injection-and-cannula-implantation-in-the-dorsal-raphe-nucleus-of-mice
#16
Patrícia A Correia, Sara Matias, Zachary F Mainen
Optogenetic methods are now widespread in neuroscience research. Here we present a detailed surgical procedure to inject adeno-associated viruses and implant optic fiber cannulas in the dorsal raphe nucleus (DRN) of living mice. Combined with transgenic mouse lines, this protocol allows specific targeting of serotonin-producing neurons in the brain. It includes fixing a mouse in a stereotaxic frame, performing a craniotomy, virus injection and fiber implantation. Animals can be later used in behavioral experiments, combined with optogenetic manipulations (Dugué et al...
September 20, 2017: Bio-protocol
https://www.readbyqxmd.com/read/29020418/striatal-n-acetylaspartate-synthetase-shati-nat8l-regulates-depression-like-behaviors-via-mglur3-mediated-serotonergic-suppression-in-mice
#17
Yoshiaki Miyamoto, Noriyuki Iegaki, Kequan Fu, Yudai Ishikawa, Kazuyuki Sumi, Sota Azuma, Kyosuke Uno, Shin-Ichi Muramatsu, Atsumi Nitta
Background: Several clinical studies have suggested that N-acetylaspartate (NAA) and N-acetylaspartylglutamate (NAAG) levels in the human brain are associated with various psychiatric disorders, including major depressive disorder. We have previously identified Shati/Nat8l, an N-acetyltransferase, in the brain using an animal model of psychosis. Shati/Nat8l synthesizes NAA from L-aspartate and acetyl-coenzyme A. Further, NAA is converted into NAAG, a neurotransmitter for metabotropic glutamate receptor 3 (mGluR3)...
August 31, 2017: International Journal of Neuropsychopharmacology
https://www.readbyqxmd.com/read/29018836/one-step-fabrication-of-bone-morphogenetic-protein-2-gene-activated-porous-poly-l-lactide-scaffold-for-bone-induction
#18
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/29018835/-13-c-31-p-mrs-metabolic-biomarkers-of-disease-progression-and-response-to-aav-delivery-of-hgaa-in-a-mouse-model-of-pompe-disease
#19
Celine Baligand, Adrian G Todd, Brittany Lee-McMullen, Ravneet S Vohra, Barry J Byrne, Darin J Falk, Glenn A Walter
The development of therapeutic clinical trials for glycogen storage disorders, including Pompe disease, has called for non-invasive and objective biomarkers. Glycogen accumulation can be measured in vivo with (13)C MRS. However, clinical implementation remains challenging due to low signal-to-noise. On the other hand, the buildup of glycolytic intermediates may be detected with (31)P MRS. We sought to identify new biomarkers of disease progression in muscle using (13)C/(31)P MRS and (1)H HR-MAS in a mouse model of Pompe disease (Gaa(-/-))...
December 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/29016934/microrna-7-upregulates-death-receptor-5-and-primes-resistant-brain-tumors-to-caspase-mediated-apoptosis
#20
Deepak Bhere, Kaoru Tamura, Hiroaki Wakimoto, Sung Hugh Choi, Benjamin Purow, Jeremy Debatisse, Khalid Shah
Background: MicroRNAs (miRs) are known to play a pivotal role in tumorigenesis, controlling cell proliferation and apoptosis. In this study, we investigated the potential of miR-7 to prime resistant tumor cells to apoptosis in glioblastoma (GBM). Methods: We created constitutive and regulatable miR-7 expression vectors and utilized pharmacological inhibition of caspases and genetic loss of function to study the effect of forced expression of miR-7 on death receptor (DR) pathways in a cohort of GBM with established resistance to tumor necrosis factor apoptosis inducing ligand (TRAIL) and in patient-derived primary GBM stem cell (GSC) lines...
July 25, 2017: Neuro-oncology
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