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https://www.readbyqxmd.com/read/29244806/cellular-selectivity-of-aav-serotypes-for-gene-delivery-in-neurons-and-astrocytes-by-neonatal-intracerebroventricular-injection
#1
Sean L Hammond, Ashley N Leek, Evan H Richman, Ronald B Tjalkens
The non-pathogenic parvovirus, adeno-associated virus (AAV), is an efficient vector for transgene expression in vivo and shows promise for treatment of brain disorders in clinical trials. Currently, there are more than 100 AAV serotypes identified that differ in the binding capacity of capsid proteins to specific cell surface receptors that can transduce different cell types and brain regions in the CNS. In the current study, multiple AAV serotypes expressing a GFP reporter (AAV1, AAV2/1, AAVDJ, AAV8, AAVDJ8, AAV9, AAVDJ9) were screened for their infectivity in both primary murine astrocyte and neuronal cell cultures...
2017: PloS One
https://www.readbyqxmd.com/read/29241457/impaired-regeneration-in-calpain-3-null-muscle-is-associated-with-perturbations-in-mtorc1-signaling-and-defective-mitochondrial-biogenesis
#2
Mehmet E Yalvac, Jakkrit Amornvit, Cilwyn Braganza, Lei Chen, Syed-Rehan A Hussain, Kimberly M Shontz, Chrystal L Montgomery, Kevin M Flanigan, Sarah Lewis, Zarife Sahenk
BACKGROUND: Previous studies in patients with limb-girdle muscular dystrophy type 2A (LGMD2A) have suggested that calpain-3 (CAPN3) mutations result in aberrant regeneration in muscle. METHODS: To gain insight into pathogenesis of aberrant muscle regeneration in LGMD2A, we used a paradigm of cardiotoxin (CTX)-induced cycles of muscle necrosis and regeneration in the CAPN3-KO mice to simulate the early features of the dystrophic process in LGMD2A. The temporal evolution of the regeneration process was followed by assessing the oxidative state, size, and the number of metabolic fiber types at 4 and 12 weeks after last CTX injection...
December 14, 2017: Skeletal Muscle
https://www.readbyqxmd.com/read/29236688/evolution-of-a-designed-protein-assembly-encapsulating-its-own-rna-genome
#3
Gabriel L Butterfield, Marc J Lajoie, Heather H Gustafson, Drew L Sellers, Una Nattermann, Daniel Ellis, Jacob B Bale, Sharon Ke, Garreck H Lenz, Angelica Yehdego, Rashmi Ravichandran, Suzie H Pun, Neil P King, David Baker
The challenges of evolution in a complex biochemical environment, coupling genotype to phenotype and protecting the genetic material, are solved elegantly in biological systems by the encapsulation of nucleic acids. In the simplest examples, viruses use capsids to surround their genomes. Although these naturally occurring systems have been modified to change their tropism and to display proteins or peptides, billions of years of evolution have favoured efficiency at the expense of modularity, making viral capsids difficult to engineer...
December 13, 2017: Nature
https://www.readbyqxmd.com/read/29234527/sphingosine-1-phosphate-receptor-1-modulates-cntf-induced-axonal-growth-and-neuroprotection-in-the-mouse-visual-system
#4
Sandrine Joly, Deniz Dalkara, Vincent Pernet
The lack of axonal regeneration and neuronal cell death causes permanent neurological deficits in the injured CNS. Using the classical CNS injury model of optic nerve crush in mice, ciliary neurotrophic factor (CNTF) was found to stimulate retinal ganglion cell (RGC) survival and axonal growth, but in an incomplete fashion. The elucidation of molecular mechanisms impairing CNTF-induced axonal regeneration is paramount to promote visual recovery. In the present study, we sought to evaluate the contribution of sphingosine 1-phosphate receptor 1 (S1PR1) to the neuroprotective and regenerative effects of CNTF...
2017: Neural Plasticity
https://www.readbyqxmd.com/read/29233995/adeno-associated-virus-serotype-rh10-is-a-useful-gene-transfer-vector-for-sensory-nerves-that-innervate-bone-in-immunodeficient-mice
#5
Sun H Park, Matthew R Eber, Shunsuke Tsuzuki, Mary E Booker, Aaron G Sunil, D Brooke Widner, Renee A Parker, Christopher M Peters, Yusuke Shiozawa
Adeno-associated virus (AAV) is frequently used to manipulate gene expression in the sensory nervous system for the study of pain mechanisms. Although some serotypes of AAV are known to have nerve tropism, whether AAV can distribute to sensory nerves that innervate the bone or skeletal tissue has not been shown. This information is crucial, since bone pain, including cancer-induced bone pain, is an area of high importance in pain biology. In this study, we found that AAVrh10 transduces neurons in the spinal cord and dorsal root ganglia of immunodeficient mice with higher efficacy than AAV2, 5, 6, 8, and 9 when injected intrathecally...
December 12, 2017: Scientific Reports
https://www.readbyqxmd.com/read/29229807/sclerostin-influences-body-composition-by-regulating-catabolic-and-anabolic-metabolism-in-adipocytes
#6
Soohyun P Kim, Julie L Frey, Zhu Li, Priyanka Kushwaha, Meredith L Zoch, Ryan E Tomlinson, Hao Da, Susan Aja, Hye Lim Noh, Jason K Kim, Mehboob A Hussain, Daniel L J Thorek, Michael J Wolfgang, Ryan C Riddle
Sclerostin has traditionally been thought of as a local inhibitor of bone acquisition that antagonizes the profound osteoanabolic capacity of activated Wnt/β-catenin signaling, but serum sclerostin levels in humans exhibit a correlation with impairments in several metabolic parameters. These data, together with the increased production of sclerostin in mouse models of type 2 diabetes, suggest an endocrine function. To determine whether sclerostin contributes to the coordination of whole-body metabolism, we examined body composition, glucose homeostasis, and fatty acid metabolism in Sost-/- mice as well as mice that overproduce sclerostin as a result of adeno-associated virus expression from the liver...
December 11, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/29227477/the-deubiquitinating-enzyme-tnfaip3-mediates-inactivation-of-hepatic-ask1-and-ameliorates-nonalcoholic-steatohepatitis
#7
Peng Zhang, Pi-Xiao Wang, Ling-Ping Zhao, Xin Zhang, Yan-Xiao Ji, Xiao-Jing Zhang, Chun Fang, Yue-Xin Lu, Xia Yang, Mao-Mao Gao, Yan Zhang, Song Tian, Xue-Yong Zhu, Jun Gong, Xin-Liang Ma, Feng Li, Zhihua Wang, Zan Huang, Zhi-Gang She, Hongliang Li
Activation of apoptosis signal-regulating kinase 1 (ASK1) in hepatocytes is a key process in the progression of nonalcoholic steatohepatitis (NASH) and a promising target for treatment of the condition. However, the mechanism underlying ASK1 activation is still unclear, and thus the endogenous regulators of this kinase remain open to be exploited as potential therapeutic targets. In screening for proteins that interact with ASK1 in the context of NASH, we identified the deubiquitinase tumor necrosis factor alpha-induced protein 3 (TNFAIP3) as a key endogenous suppressor of ASK1 activation, and we found that TNFAIP3 directly interacts with and deubiquitinates ASK1 in hepatocytes...
December 11, 2017: Nature Medicine
https://www.readbyqxmd.com/read/29226179/virus-binding-and-internalization-assay-for-adeno-associated-virus
#8
Garrett E Berry, Longping V Tse
The binding and internalization of adeno-associated virus (AAV) is an important determinant of viral infectivity and tropism. The ability to dissect these two tightly connected cellular processes would allow better understanding and provide insight on virus entry and trafficking. In the following protocol, we describe a quantitative PCR (qPCR) based method to determine the amount of vector bound to the cell surface and the amount of subsequent virus internalization based on viral genome quantification. This protocol is optimized for studying AAV...
January 20, 2017: Bio-protocol
https://www.readbyqxmd.com/read/29225043/neurogranin-in-the-nucleus-accumbens-regulates-nmda-receptor-tolerance-and-motivation-for-ethanol-seeking
#9
Ashlie N Reker, Alfredo Oliveros, John M Sullivan, Lailun Nahar, David J Hinton, Taehyun Kim, Robert C Bruner, Doo-Sup Choi, Nicholas E Goeders, Hyung W Nam
Dysfunction of N-methyl-D-aspartate receptor (NMDAR) signaling in the nucleus accumbens (NAc) has been implicated in the pathophysiology of alcohol use disorders (AUD). Neurogranin (Ng), a calmodulin-binding protein, is exclusively expressed in the post-synapse, and mediates NMDAR driven synaptic plasticity by regulating the calcium-calmodulin (Ca2+-CaM) pathway. To study the functional role of Ng in AUD, we administrated behavior tests including Pavlovian instrument transfer (PIT), operant conditioning, and rotarod test using Ng null mice (Ng-/- mice)...
December 7, 2017: Neuropharmacology
https://www.readbyqxmd.com/read/29224506/aav5-factor-viii-gene-transfer-in-severe-hemophilia-a
#10
Savita Rangarajan, Liron Walsh, Will Lester, David Perry, Bella Madan, Michael Laffan, Hua Yu, Christian Vettermann, Glenn F Pierce, Wing Y Wong, K John Pasi
Background Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia B, the large size of the factor VIII coding region has precluded improved outcomes with gene therapy in patients with hemophilia A. Methods We infused a single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding a B-domain-deleted human factor VIII (AAV5-hFVIII-SQ) in nine men with severe hemophilia A...
December 9, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29222308/hemophilia-gene-therapy-comes-of-age
#11
REVIEW
Lindsey A George
Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and our clinical trial results reporting sustained vector-derived factor IX activity well into the mild or normal range in 2016...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29221488/divergent-susceptibilities-to-aav-sacas9-grna-vector-mediated-genome-editing-in-a-single-cell-derived-cell-population
#12
Salma G Morsy, Jason M Tonne, Yaxi Zhu, Brian Lu, Karol Budzik, James W Krempski, Sherine A Ali, Mohamed A El-Feky, Yasuhiro Ikeda
OBJECTIVE: Recombinant adeno-associated virus (AAV)-based vectors are characterized by their robust and safe transgene delivery. The CRISPR/Cas9 and guide RNA (gRNA) system present a promising genome-editing platform, and a recent development of a shorter Cas9 enzyme from Staphylococcus aureus (SaCas9) allows generation of high titer single AAV vectors which carry both saCas9- and gRNA-expression cassettes. Here, we used two AAV-SaCas9 vectors with distinct GFP-targeted gRNA sequences and determined the impact of AAV-SaCas9-gRNA vector treatment in a single cell clone carrying a GFP-expression cassette...
December 8, 2017: BMC Research Notes
https://www.readbyqxmd.com/read/29217509/macrophage-associated-lipin-1-enzymatic-activity-contributes-to-modified-low-density-lipoprotein-induced-proinflammatory-signaling-and-atherosclerosis
#13
Aimee E Vozenilek, Aaron R Navratil, Jonette M Green, David T Coleman, Cassidy M R Blackburn, Alexandra C Finney, Brenna H Pearson, Roman Chrast, Brian N Finck, Ronald L Klein, A Wayne Orr, Matthew D Woolard
OBJECTIVE: Macrophage proinflammatory responses induced by modified low-density lipoproteins (modLDL) contribute to atherosclerotic progression. How modLDL causes macrophages to become proinflammatory is still enigmatic. Macrophage foam cell formation induced by modLDL requires glycerolipid synthesis. Lipin-1, a key enzyme in the glycerolipid synthesis pathway, contributes to modLDL-elicited macrophage proinflammatory responses in vitro. The objective of this study was to determine whether macrophage-associated lipin-1 contributes to atherogenesis and to assess its role in modLDL-mediated signaling in macrophages...
December 7, 2017: Arteriosclerosis, Thrombosis, and Vascular Biology
https://www.readbyqxmd.com/read/29212407/-express-rna-interference-based-functional-knockdown-of-the-voltage-gated-potassium-channel-kv7-2-in-dorsal-root-ganglion-neurons-after-in-vitro-and-in-vivo-gene-transfer-by-adeno-associated-virus-aav-vectors
#14
Markus Valdor, Anke Wagner, Viola Roehrs, Johanna Berg, Henry Fechner, Wolfgang Schroeder, Gregor Bahrenberg, Thomas Tzschentke, Thomas Christoph, Jens Kurreck
No abstract text is available yet for this article.
January 1, 2017: Molecular Pain
https://www.readbyqxmd.com/read/29212391/gene-therapy-successfully-delays-degeneration-in-a-mouse-model-of-pde6a-linked-retinitis-pigmentosa-rp-43
#15
Christian Schön, Vithiyanjali Sothilingam, Regine Mühlfriedel, Marina Garcia Garrido, Susanne C Beck, Naoyuki Tanimoto, Bernd Wissinger, François Paquet-Durand, Martin Biel, Stylianos Michalakis, Mathias W Seeliger, Rd-Cure Consortium
Retinitis pigmentosa type 43 (RP43) is a blinding disease caused by mutations in the gene for rod phosphodiesterase 6 alpha (PDE6A). The disease process begins with a dysfunction of rod photoreceptors, subsequently followed by a currently untreatable progressive degeneration of the entire outer retina. Aiming at a curative approach via PDE6A gene supplementation, we developed a novel adeno-associated viral (AAV) vector for expression of the human PDE6A cDNA under control of the human rhodopsin promotor (rAAV8...
December 7, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29212382/gene-supplementation-rescues-rod-function-and-preserves-photoreceptor-and-retinal-morphology-in-dogs-leading-the-way-towards-treating-human-pde6a-retinitis-pigmentosa
#16
Laurence M Occelli, Christian Schön, Mathias W Seeliger, Martin Biel, Stylianos Michalakis, Simon Petersen-Jones, Rd-Cure Consortium
Mutations in the phosphodiesterase 6A gene (PDE6A) result in retinitis pigmentosa type 43 (RP43) and are responsible for about 4% of autosomal recessive RP. There is currently no treatment for this blinding condition. The aim of this project was to use a large animal model to test a gene supplementation viral vector designed to be translated for use in a clinical trial for the treatment of RP43. Seven Pde6a<sup>-/-</sup> puppies were given subretinal injections of an adeno-associated viral vector serotype 2/8 delivering human PDE6A cDNA under control of a short rhodopsin promoter (AAV8-PDE6A)...
December 6, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/29211814/overexpression-of-transmembrane-protein-168-in-the-mouse-nucleus-accumbens-induces-anxiety-and-sensorimotor-gating-deficit
#17
Kequan Fu, Yoshiaki Miyamoto, Kazuyuki Sumi, Eriko Saika, Shin-Ichi Muramatsu, Kyosuke Uno, Atsumi Nitta
Transmembrane protein 168 (TMEM168) comprises 697 amino acid residues, including some putative transmembrane domains. It is reported that TMEM168 controls methamphetamine (METH) dependence in the nucleus accumbens (NAc) of mice. Moreover, a strong link between METH dependence-induced adaptive changes in the brain and mood disorders has been evaluated. In the present study, we investigated the effects of accumbal TMEM168 in a battery of behavioral paradigms. The adeno-associated virus (AAV) Tmem168 vector was injected into the NAc of C57BL/6J mice (NAc-TMEM mice)...
2017: PloS One
https://www.readbyqxmd.com/read/29211678/hemophilia-b-gene-therapy-with-a-high-specific-activity-factor-ix-variant
#18
Lindsey A George, Spencer K Sullivan, Adam Giermasz, John E J Rasko, Benjamin J Samelson-Jones, Jonathan Ducore, Adam Cuker, Lisa M Sullivan, Suvankar Majumdar, Jerome Teitel, Catherine E McGuinn, Margaret V Ragni, Alvin Y Luk, Daniel Hui, J Fraser Wright, Yifeng Chen, Yun Liu, Katie Wachtel, Angela Winters, Stefan Tiefenbacher, Valder R Arruda, Johannes C M van der Loo, Olga Zelenaia, Daniel Takefman, Marcus E Carr, Linda B Couto, Xavier M Anguela, Katherine A High
BACKGROUND: The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia. METHODS: We infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX-R338L) transgene at a dose of 5×1011 vector genomes per kilogram of body weight in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value...
December 7, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29208777/short-a%C3%AE-peptides-attenuate-a%C3%AE-42-toxicity-in-vivo
#19
Brenda D Moore, Jason Martin, Lorena de Mena, Jonatan Sanchez, Pedro E Cruz, Carolina Ceballos-Diaz, Thomas B Ladd, Yong Ran, Yona Levites, Thomas L Kukar, Justin J Kurian, Robert McKenna, Edward H Koo, David R Borchelt, Christopher Janus, Diego Rincon-Limas, Pedro Fernandez-Funez, Todd E Golde
Processing of amyloid-β (Aβ) precursor protein (APP) by γ-secretase produces multiple species of Aβ: Aβ40, short Aβ peptides (Aβ37-39), and longer Aβ peptides (Aβ42-43). γ-Secretase modulators, a class of Alzheimer's disease therapeutics, reduce production of the pathogenic Aβ42 but increase the relative abundance of short Aβ peptides. To evaluate the pathological relevance of these peptides, we expressed Aβ36-40 and Aβ42-43 in Drosophila melanogaster to evaluate inherent toxicity and potential modulatory effects on Aβ42 toxicity...
December 5, 2017: Journal of Experimental Medicine
https://www.readbyqxmd.com/read/29204900/molecular-design-for-recombinant-adeno-associated-virus-raav-vector-production
#20
REVIEW
Juan Jose Aponte-Ubillus, Daniel Barajas, Joseph Peltier, Cameron Bardliving, Parviz Shamlou, Daniel Gold
Recombinant adeno-associated virus (rAAV) vectors are increasingly popular tools for gene therapy applications. Their non-pathogenic status, low inflammatory potential, availability of viral serotypes with different tissue tropisms, and prospective long-lasting gene expression are important attributes that make rAAVs safe and efficient therapeutic options. Over the last three decades, several groups have engineered recombinant AAV-producing platforms, yielding high titers of transducing vector particles. Current specific productivity yields from different platforms range from 103 to 105 vector genomes (vg) per cell, and there is an ongoing effort to improve vector yields in order to satisfy high product demands required for clinical trials and future commercialization...
December 4, 2017: Applied Microbiology and Biotechnology
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