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adeno-associated viral

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https://www.readbyqxmd.com/read/28726522/steerable-induction-of-the-thymosin-%C3%A3-4-mrtf-a-pathway-via-aav-based-overexpression-induces-therapeutic-neovascularization
#1
Tilman Ziegler, Markus Kraus, Wira Husada, Florian Gesenhues, Qui Jiang, Olaf Pinkenburg, Teresa Trenkwalder, Karl-Ludwig Laugwitz, Ferdinand le Noble, Christian Weber, Christian Kupatt, Rabea Hinkel
Viral vectors have been frequently used in a variety of preclinical animal models to deliver genetic constructs into tissues. Among the vectors used, adeno-associated viral vectors (AAVs) may be targeted to specific tissues, depending on the serotype used. Moreover, they show robust expression for prolonged periods of time and have a low immunogenic potential. Furthermore, AAVs, unlike other vector systems, only display a low rate of genomic integration. However, to ensure efficient transgene production, expression is typically driven by constitutively active promoters, such as the CMV promotor...
July 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28723575/crispr-mediated-integration-of-large-gene-cassettes-using-aav-donor-vectors
#2
Rasmus O Bak, Matthew H Porteus
The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA during homologous recombination (HR). However, the maximum AAV packaging capacity of ∼4.5 kb limits the donor size. Here, we overcome this constraint by showing that two co-transduced AAV vectors can serve as donors during consecutive HR events for the integration of large transgenes. Importantly, the method involves a single-step procedure applicable to primary cells with relevance to therapeutic genome editing...
July 18, 2017: Cell Reports
https://www.readbyqxmd.com/read/28720467/intradermal-immunization-with-raav1-vector-induces-robust-memory-cd8-t-cell-responses-independently-of-transgene-expression-in-dcs
#3
Alexandre Ghenassia, David-Alexandre Gross, Stéphanie Lorain, Fabiola Tros, Dominique Urbain, Sofia Benkhelifa-Ziyyat, Alain Charbit, Jean Davoust, Pascal Chappert
Recombinant adeno-associated viral (rAAV) vectors exhibit interesting properties as vaccine carriers for their ability to induce long-lasting antibody responses. However, rAAV-based vaccines have been suggested to trigger functionally impaired long-term memory CD8(+) T cell responses, in part due to poor dendritic cell (DC) transduction. Such results, albeit limited to intramuscular immunization, undermined the use of rAAV as vaccine vehicles against intracellular pathogens. We report here that intradermal immunization with a model rAAV2/1-based vaccine drives the development of bona fide long-term memory CD8(+) T cell responses...
July 15, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28713896/an-optimized-gene-transfection-system-in-weri-rb1-cells
#4
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
#5
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/28708260/effect-of-optogenetic-manipulation-of-accumbal-medium-spiny-neurons-expressing-dopamine-d2-receptors-in-cocaine-induced-behavioral-sensitization
#6
Byeong Jun Kang, Shelly Sooyun Song, Lei Wen, Ki-Pyo Hong, George J Augustine, Ja-Hyun Baik
Repetitive exposure to addictive drugs causes synaptic modification in the mesocorticolimbic dopamine (DA) system. Dopamine D1 receptors (D1R) or D2 receptors (D2R) expressed in the medium spiny neurons (MSNs) of the nucleus accumbens (NAc) play critical roles in the control of addictive behaviors. Optogenetic activation of D2R-expressing MSNs (D2R-MSNs) in the NAc previously demonstrated that these neurons play a key role in withdrawal-induced plasticity. Here, we examined the effect of optogenetic inhibition of D2R-MSNs in the NAc on cocaine-induced behavioral sensitization...
July 14, 2017: European Journal of Neuroscience
https://www.readbyqxmd.com/read/28706756/impact-of-vital-dyes-on-cell-viability-and-transduction-efficiency-of-aav-vectors-used-in-retinal-gene-therapy-surgery-an-in-vitro-and-in-vivo-analysis
#7
Anna P Salvetti, Maria I Patrício, Alun R Barnard, Harry O Orlans, Doron G Hickey, Robert E MacLaren
PURPOSE: Treatment of inherited retinal degenerations using adeno-associated viral (AAV) vectors involves delivery by subretinal injection. In the latter stages, alteration of normal anatomy may cause difficulty in visualizing the retinotomy, retinal detachment extension, and vector diffusion. Vital dyes may be useful surgical adjuncts, but their safety and impact on AAV transduction are largely unknown. METHODS: The effects of Sodium Fluorescein (SF), Membrane Blue (MB), and Membrane Blue Dual (DB) at a range of dilutions were assessed on human embryonic kidney cells in vitro using an AAV2-green fluorescent protein (GFP) reporter at different multiplicities of infection...
July 2017: Translational Vision Science & Technology
https://www.readbyqxmd.com/read/28704696/the-function-of-dna-binding-protein-nucleophosmin-in-aav-replication
#8
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/28698564/homer-binds-to-orai1-and-trpc-channels-in-the-neointima-and-regulates-vascular-smooth-muscle-cell-migration-and-proliferation
#9
Shuping Jia, Miguel Rodriguez, Arthur G Williams, Joseph P Yuan
The molecular components of store-operated Ca(2+) influx channels (SOCs) in proliferative and migratory vascular smooth muscle cells (VSMCs) are quite intricate with many channels contributing to SOCs. They include the Ca(2+)-selective Orai1 and members of the transient receptor potential canonical (TRPC) channels, which are activated by the endoplasmic reticulum Ca(2+) sensor STIM1. The scaffolding protein Homer assembles SOC complexes, but its role in VSMCs is not well understood. Here, we asked whether these SOC components and Homer1 are present in the same complex in VSMCs and how Homer1 contributes to VSMC SOCs, proliferation, and migration leading to neointima formation...
July 11, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28696391/site-specific-pegylated-adeno-associated-viruses-with-increased-serum-stability-and-reduced-immunogenicity
#10
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/28679762/aav-serotypes-have-distinctive-interactions-with-domains-of-the-cellular-receptor-aavr
#11
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/28672171/host-determinants-of-adeno-associated-viral-vector-entry
#12
REVIEW
Sirika Pillay, Jan E Carette
Viral vectors based on adeno-associated virus (AAV) are leading candidates for therapeutic gene delivery. Understanding rate-limiting steps in the entry of AAV vectors may be used in a rational approach to improve efficiency and specificity of transduction. This review describes our current understanding of AAV entry, a key step during infection. We discuss the identity and functions of AAV receptors and attachment factors, including the recently discovered multi-serotype receptor AAVR. We further provide an overview of other host factors that act during the trafficking stage of AAV vector transduction...
June 30, 2017: Current Opinion in Virology
https://www.readbyqxmd.com/read/28669112/adeno-associated-virus-aav-as-a-vector-for-gene-therapy
#13
REVIEW
Michael F Naso, Brian Tomkowicz, William L Perry, William R Strohl
There has been a resurgence in gene therapy efforts that is partly fueled by the identification and understanding of new gene delivery vectors. Adeno-associated virus (AAV) is a non-enveloped virus that can be engineered to deliver DNA to target cells, and has attracted a significant amount of attention in the field, especially in clinical-stage experimental therapeutic strategies. The ability to generate recombinant AAV particles lacking any viral genes and containing DNA sequences of interest for various therapeutic applications has thus far proven to be one of the safest strategies for gene therapies...
July 1, 2017: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
https://www.readbyqxmd.com/read/28667125/induction-of-eao-in-mice-responses-to-elevated-circulating-levels-of-the-follistatin
#14
Nour Nicolas, Julie A Muir, Susan Hayward, Justin L Chen, Peter G Stanton, Paul Gregorevic, David M de Kretser, Kate L Loveland, Sudhanshu Bhushan, Andreas Meinhardt, Monika Fijak, Mark P Hedger
Experimental autoimmune orchitis (EAO) is a rodent model of chronic testicular inflammation that mimics the pathology observed in some types of human infertility. In a previous study, testicular expression of the inflammatory/immunoregulatory cytokine, activin A, was elevated in adult mice during the onset of EAO, indicating a potential role in regulation of the disease. Consequently, we examined the development of EAO in mice with elevated levels of follistatin, an endogenous activin antagonist, as a potential therapeutic approach to testicular inflammation...
June 30, 2017: Reproduction: the Official Journal of the Society for the Study of Fertility
https://www.readbyqxmd.com/read/28667090/lentiviral-vectors-escape-innate-sensing-but-trigger-p53-in-human-hematopoietic-stem-and-progenitor-cells
#15
Francesco Piras, Michela Riba, Carolina Petrillo, Dejan Lazarevic, Ivan Cuccovillo, Sara Bartolaccini, Elia Stupka, Bernhard Gentner, Davide Cittaro, Luigi Naldini, Anna Kajaste-Rudnitski
Clinical application of lentiviral vector (LV)-based hematopoietic stem and progenitor cells (HSPC) gene therapy is rapidly becoming a reality. Nevertheless, LV-mediated signaling and its potential functional consequences on HSPC biology remain poorly understood. We unravel here a remarkably limited impact of LV on the HSPC transcriptional landscape. LV escaped innate immune sensing that instead led to robust IFN responses upon transduction with a gamma-retroviral vector. However, reverse-transcribed LV DNA did trigger p53 signaling, activated also by non-integrating Adeno-associated vector, ultimately leading to lower cell recovery ex vivo and engraftment in vivo These effects were more pronounced in the short-term repopulating cells while long-term HSC frequencies remained unaffected...
June 30, 2017: EMBO Molecular Medicine
https://www.readbyqxmd.com/read/28666837/operant-responding-for-optogenetic-excitation-of-ldtg-inputs-to-the-vta-requires-d1-and-d2-dopamine-receptor-activation-in-the-nacc
#16
Stephan Steidl, Shannon O'Sullivan, Dustin Pilat, Nancy Bubula, Jason Brown, Paul Vezina
Behavioral studies in rats and mice indicate that laterodorsal tegmental nucleus (LDTg) inputs to the ventral tegmental area (VTA) importantly contribute to reward function. Further evidence from anesthetized rat and mouse preparations suggests that these LTDg inputs may exert this effect by regulating mesolimbic dopamine (DA) signaling. Direct evidence supporting this possibility remains lacking however. To address this lack, rat LDTg neurons were transfected with adeno-associated viral vectors encoding channelrhodopsin2 and eYFP (ChR2) or eYFP alone (eYFP) and rats were subsequently trained to lever press for intracranial self-stimulation (ICSS) of the inputs of these neurons to the VTA...
June 27, 2017: Behavioural Brain Research
https://www.readbyqxmd.com/read/28665147/s-mar-element-facilitates-episomal-long-term-persistence-of-adeno-associated-viral-aav-vector-genomes-in-proliferating-cells
#17
Claudia Hagedorn, Maria Schnödt, Philip Boehme, Heba Abdelrazik, Hans Lipps, Hildegard Büning
Adeno-associated viral (AAV) vectors are one of the most frequently applied gene transfer system in research and human clinical trials. Since AAV vectors do not possess an integrase activity, application is restricted to terminally differentiated tissues if transgene expression is required long-term. To overcome this limitation and to generate AAV vectors that persist episomally in dividing cells, we equipped AAV vector genomes with an autonomous replicating unit (Scaffold/matrix attachment region (S/MAR))...
June 30, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28662750/viral-vector-biosafety-in-laboratory-animal-research
#18
Dalis E Collins, Jon D Reuter, Howard G Rush, Jason S Villano
Viral vector research presents unique occupational health and safety challenges to institutions due to the rapid development of both in vivo and in vitro gene-editing technologies. Risks to human and animal health make it incumbent on institutions to appropriately evaluate viral vector usage in research on the basis of available information and governmental regulations and guidelines. Here we review the factors related to risk assessment regarding viral vector usage in animals and the relevant regulatory documents associated with this research, and we highlight the most commonly used viral vectors in research today...
June 1, 2017: Comparative Medicine
https://www.readbyqxmd.com/read/28660346/adeno-associated-viral-gene-therapy-for-mucopolysaccharidoses-exhibiting-neurodegeneration
#19
REVIEW
Adeline A Lau, Kim M Hemsley
The mucopolysaccharidoses (MPS) are a subgroup of lysosomal storage disorders that are caused by mutations in the genes involved in glycosaminoglycan breakdown. Multiple organs and tissues are affected, including the central nervous system. At present, hematopoietic stem cell transplantation and enzyme replacement therapies are approved for some of the (non-neurological) MPS. Treatments that effectively ameliorate the neurological aspects of the disease are being assessed in clinical trials. This review will focus on the recent outcomes and planned viral vector-mediated gene therapy clinical trials, and the pre-clinical data that supported these studies, for MPS-I (Hurler/Scheie syndrome), MPS-II (Hunter syndrome), and MPS-IIIA and -IIIB (Sanfilippo syndrome)...
June 29, 2017: Journal of Molecular Medicine: Official Organ of the "Gesellschaft Deutscher Naturforscher und Ärzte"
https://www.readbyqxmd.com/read/28659483/human-bocavirus-1-is-a-novel-helper-for-adeno-associated-virus-replication
#20
Zekun Wang, Xuefeng Deng, Wei Zou, John F Engelhardt, Ziying Yan, Jianming Qiu
Human bocavirus 1 (HBoV1) is an autonomous parvovirus that infects well-differentiated primary human airway epithelia (HAE) in vitro. In human embryonic kidney (HEK) 293 cells, transfection of a duplex HBoV1 genome initiates viral DNA replication and produces progeny virions that are infectious in HAE. HBoV1 takes advantage of signaling pathways in the DNA damage response for efficient genome amplification in both well-differentiated (non-dividing) HAE and dividing HEK293 cells. On the other hand, adeno-associated virus 2 (AAV2) is a helper-dependent dependoparvovirus, and productive AAV2 replication requires co-infection with a helper virus (e...
June 28, 2017: Journal of Virology
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