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adeno associated virus

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https://www.readbyqxmd.com/read/28715454/reduction-of-cav1-3-channels-in-dorsal-hippocampus-impairs-the-development-of-dentate-gyrus-newborn-neurons-and-hippocampal-dependent-memory-tasks
#1
Su-Hyun Kim, Ye-Ryoung Park, Boyoung Lee, Byungil Choi, Hyun Kim, Chong-Hyun Kim
Cav1.3 has been suggested to mediate hippocampal neurogenesis of adult mice and contribute to hippocampal-dependent learning and memory processes. However, the mechanism of Cav1.3 contribution in these processes is unclear. Here, roles of Cav1.3 of mouse dorsal hippocampus during newborn cell development were examined. We find that knock-out (KO) of Cav1.3 resulted in the reduction of survival of newborn neurons at 28 days old after mitosis. The retroviral eGFP expression showed that both dendritic complexity and the number and length of mossy fiber bouton (MFB) filopodia of newborn neurons at ≥ 14 days old were significantly reduced in KO mice...
2017: PloS One
https://www.readbyqxmd.com/read/28714989/correction-of-a-splicing-defect-in-a-mouse-model-of-congenital-muscular-dystrophy-type-1a-using-a-homology-directed-repair-independent-mechanism
#2
Dwi U Kemaladewi, Eleonora Maino, Elzbieta Hyatt, Huayun Hou, Maylynn Ding, Kara M Place, Xinyi Zhu, Prabhpreet Bassi, Zahra Baghestani, Amit G Deshwar, Daniele Merico, Hui Y Xiong, Brendan J Frey, Michael D Wilson, Evgueni A Ivakine, Ronald D Cohn
Splice-site defects account for about 10% of pathogenic mutations that cause Mendelian diseases. Prevalence is higher in neuromuscular disorders (NMDs), owing to the unusually large size and multi-exonic nature of genes encoding muscle structural proteins. Therapeutic genome editing to correct disease-causing splice-site mutations has been accomplished only through the homology-directed repair pathway, which is extremely inefficient in postmitotic tissues such as skeletal muscle. Here we describe a strategy using nonhomologous end-joining (NHEJ) to correct a pathogenic splice-site mutation...
July 17, 2017: Nature Medicine
https://www.readbyqxmd.com/read/28713896/an-optimized-gene-transfection-system-in-weri-rb1-cells
#3
Ying Liu, Zhigang Fan, Kang Li, Fei Deng, Yunfan Xiong, Meixin Liang, Jian Ge
The pathogenesis of Rb1 gene inactivation indicates that gene therapy could be a promising treatment for retinoblastoma. An appropriate gene transfer system is the basis for successful gene therapy; however, little attention has been given to an effective gene transfer system for retinoblastoma therapy in previous studies. This study was designed to provide an optimized transgene system for WERI‑Rb1 cells (W-RBCs). Green fluorescent protein (GFP) was adopted as a reporter. Four classic viral vectors based on retroviruses, recombinant adeno-associated viruses (rAAV2, rAAV2/1), lentiviruses (LVs) and a novel non-viral vector X-treme HP reagent were adopted for W-RBC gene transfection...
July 6, 2017: International Journal of Molecular Medicine
https://www.readbyqxmd.com/read/28710345/deletion-of-the-b-b-and-c-c-regions-of-inverted-terminal-repeats-reduces-raav-productivity-but-increases-transgene-expression
#4
Qingzhang Zhou, Wenhong Tian, Chunguo Liu, Zhonghui Lian, Xiaoyan Dong, Xiaobing Wu
Inverted terminal repeats (ITRs) of the adeno-associated virus (AAV) are essential for rescue, replication, packaging, and integration of the viral genome. While ITR mutations have been identified in previous reports, we designed a new truncated ITR lacking the B-B' and C-C' regions named as ITRΔBC and investigated its effects on viral genome replication, packaging, and expression of recombinant AAV (rAAV). The packaging ability was compared between ITRΔBC rAAV and wild-type (wt) ITR rAAV. Our results showed the productivity of ITRΔBC rAAV was reduced 4-fold, which is consistent with the 8-fold decrease in the replication of viral genomic DNA of ITRΔBC rAAV compared with wt ITR rAAV...
July 14, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28707952/systemic-delivery-of-dysferlin-overlap-vectors-provides-long-term-functional-improvement-for-dysferlinopathy
#5
Rachael A Potter, Danielle A Griffin, Patricia C Sondergaard, Ryan W Johnson, Eric R Pozsgai, Kristin N Heller, Ellyn L Peterson, Kimmo K Lehtimaki, Hillarie P Windish, Plavi J Mittal, Doug E Albrecht, Jerry R Mendell, Louise R Rodino-Klapac
Dysferlinopathies comprise a family of disorders caused by mutations in the dysferlin (DYSF) gene leading to a progressive dystrophy characterized by chronic muscle fiber loss, fat replacement and fibrosis. To correct the underlying histopathology and function, expression of full-length DYSF is required. We have developed dual adeno-associated virus vectors defined by a region of homology to serve as a substrate for reconstitution of the full 6.5 kb dysferlin cDNA. Our previous work studied the efficacy of this treatment through intramuscular and regional delivery routes...
July 14, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28704696/the-function-of-dna-binding-protein-nucleophosmin-in-aav-replication
#6
Stifani Satkunanathan, Robin Thorpe, Yuan Zhao
Adeno-associated viruses (AAV) contain minimal viral proteins necessary for their replication. During virus assembly, AAV acquire, inherently and submissively, various cellular proteins. Our previous studies identified the association of AAV vectors with the DNA binding protein nucleophosmin (NPM1). Nucleophosmin has been reported to enhance AAV infection by mobilizing AAV capsids into and out of the nucleolus, indicating the importance of NPM1 in the AAV life cycle; however the role of NPM1 in AAV production remains unknown...
July 10, 2017: Virology
https://www.readbyqxmd.com/read/28702474/targeting-visceral-fat-by-intraperitoneal-delivery-of-novel-aav-serotype-vector-restricting-off-target-transduction-in-liver
#7
Wei Huang, Xianglan Liu, Nicholas J Queen, Lei Cao
It is challenging to genetically manipulate fat in adults. We demonstrate that intraperitoneal (i.p.) injection of an engineered adeno-associated virus (AAV) serotype Rec2 leads to high transduction of multiple visceral fat depots at a dose of 1 to 2 orders lower than commonly used doses for systemic gene delivery. To target adipose tissue, we develop a single AAV vector harboring two expression cassettes: one using the CBA promoter to drive transgene expression and one using the liver-specific albumin promoter to drive a microRNA-targeting WPRE sequence that only exists in this AAV vector...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28702323/aav-mediated-pancreatic-overexpression-of-igf1-counteracts-progression-to-autoimmune-diabetes-in-mice
#8
Cristina Mallol, Estefania Casana, Veronica Jimenez, Alba Casellas, Virginia Haurigot, Claudia Jambrina, Victor Sacristan, Meritxell Morró, Judith Agudo, Laia Vilà, Fatima Bosch
OBJECTIVE: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches, focusing on preserving/expanding β-cell mass and/or blocking the autoimmune process that destroys islets, should be developed. The main objective of this work was to test in non-obese diabetic (NOD) mice, which spontaneously develop autoimmune diabetes, the effects of local expression of Insulin-like growth factor 1 (IGF1), a potent mitogenic and pro-survival factor for β-cells with immunomodulatory properties...
July 2017: Molecular Metabolism
https://www.readbyqxmd.com/read/28700093/interleukin-10-release-from-astrocytes-suppresses-neuronal-apoptosis-via-the-tlr2-nf%C3%AE%C2%BAb-pathway-in-a-neonatal-rat-model-of-hypoxic-ischemic-brain-damage
#9
Mu Lan He, Ze Yu Lv, Xia Shi, Ting Yang, Yun Zhang, Ting-Yu Li, Jie Chen
The biological function of Interleukin-10 (IL-10) and the relationship between IL-10 secretion and the Toll-like receptor 2 (TLR2) expression levels in the central nervous system following hypoxic-ischemic brain damage (HIBD) are poorly understood. Here, we intend to elucidate the biological function and mechanism of IL-10 secretion following HIBD. In the current study, we used a neonatal rat model of HIBD and found that rats injected with adeno-associated virus (AAV)-IL-10-shRNA (short hairpin RNA) exhibited partially impaired learning and memory function compared to rats administered AAV-control-shRNA...
July 12, 2017: Journal of Neurochemistry
https://www.readbyqxmd.com/read/28700081/spinal-nociceptive-circuit-analysis-with-recombinant-adeno-associated-viruses-the-impact-of-serotypes-and-promoters
#10
Karen Haenraets, Edmund Foster, Helge Johannssen, Vinnie Kandra, Noemie Frezel, Timothy Steffen, Valeria Jaramillo, Jean-Charles Paterna, Hanns Ulrich Zeilhofer, Hendrik Wildner
Recombinant adeno-associated virus (rAAV) vector-mediated gene transfer into genetically defined neuron subtypes has become a powerful tool to study the neuroanatomy of neuronal circuits in the brain and to unravel their functions. More recently this methodology has also become popular for the analysis of spinal cord circuits. To date a variety of naturally occurring AAV serotypes and genetically modified capsid variants are available but transduction efficiency in spinal neurons, target selectivity and the ability for retrograde tracing are only incompletely characterized...
July 12, 2017: Journal of Neurochemistry
https://www.readbyqxmd.com/read/28697556/probdnf-accelerates-brain-amyloid-%C3%AE-deposition-and-learning-and-memory-impairment-in-appsweps1de9-transgenic-mice
#11
Jia Chen, Tao Zhang, Shusheng Jiao, Xinfu Zhou, Jinhua Zhong, Yanjiang Wang, Juan Liu, Juan Deng, Shuiping Wang, Zhiqiang Xu
BACKGROUND: Alzheimer's disease (AD) is pathologically known for the amyloid-β (Aβ) deposition, neurofibrillary tangles, and neuronal loss in the brain. The precursor of brain-derived neurotrophic factor (proBDNF) before proteolysis has opposing functions to its mature form in neuronal survival and neurite growth. However, the role of proBDNF in the pathogenesis of AD remains unclear. OBJECTIVE: To investigate the effects of proBDNF on neurons in vitro, and on learning and memory impairment and brain Aβ production in a transgenic AD mouse model (APPswePS1dE9)...
July 1, 2017: Journal of Alzheimer's Disease: JAD
https://www.readbyqxmd.com/read/28696391/site-specific-pegylated-adeno-associated-viruses-with-increased-serum-stability-and-reduced-immunogenicity
#12
Tianzhuo Yao, Xueying Zhou, Chuanling Zhang, Xiaojuan Yu, Zhenyu Tian, Lihe Zhang, Demin Zhou
Adeno-associated virus (AAV) is one of the most extensively studied and utilized viral vectors in clinical gene transfer research. However, the serum instability and immunogenicity of AAV vectors significantly limit their application. Here, we endeavored to overcome these limitations by developing a straightforward approach for site-specific PEGylation of AAV via genetic code expansion. This technique includes incorporation of the azide moiety into the AAV capsid protein followed by orthogonal and stoichiometric conjugation of a variety of polyethylene glycols (PEGs) through click chemistry...
July 11, 2017: Molecules: a Journal of Synthetic Chemistry and Natural Product Chemistry
https://www.readbyqxmd.com/read/28688268/tfeb-mediated-activation-of-the-lysosome-autophagy-system-affects-the-transduction-efficiency-of-adeno-associated-virus-2
#13
Lauren Popp, Eric Gomez, Whitney Orji, Michelle Ho, Junghae Suh, Laura Segatori
Adeno-associated virus (AAV)-mediated gene transfer is an appealing therapeutic option due to AAV's safety profile. Effective delivery of AAV's genetic cargo to the nucleus, however, requires evasion of host cell barriers, including cellular clearance mechanisms mediated by the lysosome-autophagy system. We used AAV serotype 2 to monitor the autophagic response to cellular internalization of AAV and to characterize the effect of AAV-induced activation of autophagy on transgene expression. We found AAV2 internalization to induce activation of transcription factor EB, a master regulator of autophagy and lysosomal biogenesis, and upregulation of the lysosome-autophagy system...
July 5, 2017: Virology
https://www.readbyqxmd.com/read/28687495/extracellular-vesicles-novel-promising-delivery-systems-for-therapy-of-brain-diseases
#14
REVIEW
David Rufino-Ramos, Patrícia R Albuquerque, Vitor Carmona, Rita Perfeito, Rui Jorge Nobre, Luis Pereira de Almeida
Extracellular vesicles (EVs) are cell-derived membrane vesicles virtually secreted by all cells, including brain cells. EVs are a major term that includes apoptotic bodies, microvesicles and exosomes. The release of EVs has been recognized as an important modulator in cross-talking between neurons, astrocytes, microglia and oligodendrocytes, not only in central nervous system (CNS) physiology but also in neurodegenerative and neuroinflammatory disease states as well as in brain tumors, such as glioma. EVs are able to cross the blood brain barrier (BBB), spread to body fluids and reach distant tissues...
July 4, 2017: Journal of Controlled Release: Official Journal of the Controlled Release Society
https://www.readbyqxmd.com/read/28683532/recombinant-adeno-associated-virus-expressing-truncated-ik-cytokine-diminishes-the-symptoms-of-inflammatory-arthritis
#15
Seulgi Choi, Hyelim Park, Marstella Minelko, Eun-Kyung Kim, Mi-Ra Cho, Jae-Hwan Nam
IK can downregulate interferon (IFN)-γ-induced major histocompatibility complex (MHC) class II expression through the MHC class II transactivator, which suggests that IK can inhibit the interactions between immune cells. We delivered adeno-associated virus serotype 2 (AAV2) encoding the genes for truncated IK (tIK) or green fluorescent protein (GFP) to DBA1/J mice via intravenous injection. Seven weeks after injection, collagen-induced arthritis (CIA) was induced in the AAV2 treated mice. AAV2-tIK injection reduced the severity of arthritis and the percentage of pathogenic Th17 cells compared with AAV2-GFP injection...
July 7, 2017: Journal of Microbiology and Biotechnology
https://www.readbyqxmd.com/read/28679762/aav-serotypes-have-distinctive-interactions-with-domains-of-the-cellular-receptor-aavr
#16
Sirika Pillay, Wei Zou, Fang Cheng, Andreas S Puschnik, Nancy L Meyer, Safder S Ganaie, Xuefeng Deng, Jonathan E Wosen, Omar Davulcu, Ziying Yan, John F Engelhardt, Kevin E Brown, Michael S Chapman, Jianming Qiu, Jan E Carette
Adeno-associated virus (AAV) entry is determined by its interactions with specific surface glycans and proteinaceous receptor(s). Adeno-associated virus receptor (AAVR; also named KIAA0319L) is an essential cellular receptor required for the transduction of vectors derived from multiple AAV serotypes including the evolutionary distant serotypes, AAV2 and AAV5. Here, we further biochemically characterize the AAV-AAVR interaction and define the domains within the ectodomain of AAVR that facilitate this interaction...
July 5, 2017: Journal of Virology
https://www.readbyqxmd.com/read/28679290/gene-therapy-using-a-minicep290-fragment-delays-photoreceptor-degeneration-in-a-mouse-model-of-leber-congenital-amaurosis-lca
#17
Wei Zhang, Linjing Li, Qin Su, Guangping Gao, Hemant Khanna
Mutations in the cilia-centrosomal protein CEP290 are most frequently observed in autosomal recessive childhood blindness disorder Leber congenital amaurosis (LCA). No treatment or cure currently exists for this disorder. The Cep290rd16 (retinal degeneration 16) (a mouse model of LCA) carries a mutation in the Cep290 gene, which leads to shorter cilia formation and defective photoreceptor structure and function. A roadblock to developing a gene replacement strategy for CEP290 using conventional Adeno-associated Virus (AAV) vectors is its large size...
July 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28677202/dreadd-induced-silencing-of-the-medial-amygdala-reduces-the-preference-for-male-pheromones-and-the-expression-of-lordosis-in-estrous-female-mice
#18
Elizabeth A McCarthy, Arman Maqsudlu, Matthew Bass, Sofia Georghiou, James A Cherry, Michael J Baum
Sexually naïve estrous female mice seek out male urinary pheromones; however, they initially display little receptive (lordosis) behavior in response to male mounts. Vomeronasal - accessory olfactory bulb inputs to the medial amygdala (Me) regulate courtship in female rodents. We used a reversible inhibitory chemogenetic technique (Designer Receptors Exclusively Activated by Designer Drugs; DREADDs) to assess the contribution of Me signaling to females' preference for male pheromones and improvement in receptivity normally seen with repeated testing...
July 5, 2017: European Journal of Neuroscience
https://www.readbyqxmd.com/read/28676737/gene-therapy-in-a-large-animal-model-of-pde6a-retinitis-pigmentosa
#19
Freya M Mowat, Laurence M Occelli, Joshua T Bartoe, Kristen J Gervais, Ashlee R Bruewer, Janice Querubin, Astra Dinculescu, Sanford L Boye, William W Hauswirth, Simon M Petersen-Jones
Despite mutations in the rod phosphodiesterase 6-alpha (PDE6A) gene being well-recognized as a cause of human retinitis pigmentosa, no definitive treatments have been developed to treat this blinding disease. We performed a trial of retinal gene augmentation in the Pde6a mutant dog using Pde6a delivery by capsid-mutant adeno-associated virus serotype 8, previously shown to have a rapid onset of transgene expression in the canine retina. Subretinal injections were performed in 10 dogs at 29-44 days of age, and electroretinography and vision testing were performed to assess functional outcome...
2017: Frontiers in Neuroscience
https://www.readbyqxmd.com/read/28673981/synergistic-effects-of-treating-the-spinal-cord-and-brain-in-cln1-disease
#20
Charles Shyng, Hemanth R Nelvagal, Joshua T Dearborn, Jaana Tyynelä, Robert E Schmidt, Mark S Sands, Jonathan D Cooper
Infantile neuronal ceroid lipofuscinosis (INCL, or CLN1 disease) is an inherited neurodegenerative storage disorder caused by a deficiency of the lysosomal enzyme palmitoyl protein thioesterase 1 (PPT1). It was widely believed that the pathology associated with INCL was limited to the brain, but we have now found unexpectedly profound pathology in the human INCL spinal cord. Similar pathological changes also occur at every level of the spinal cord of PPT1-deficient (Ppt1(-/-) ) mice before the onset of neuropathology in the brain...
July 3, 2017: Proceedings of the National Academy of Sciences of the United States of America
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