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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
#1
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/29211814/overexpression-of-transmembrane-protein-168-in-the-mouse-nucleus-accumbens-induces-anxiety-and-sensorimotor-gating-deficit
#2
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/29208777/short-a%C3%AE-peptides-attenuate-a%C3%AE-42-toxicity-in-vivo
#3
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
#4
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
https://www.readbyqxmd.com/read/29204184/viral-cre-loxp-tools-aid-genome-engineering-in-mammalian-cells
#5
Ranjita Sengupta, Amy Mendenhall, Nandita Sarkar, Chandreyee Mukherjee, Amirali Afshari, Joseph Huang, Biao Lu
Background: Targeted nucleases have transformed genome editing technology, providing more efficient methods to make targeted changes in mammalian genome. In parallel, there is an increasing demand of Cre-LoxP technology for complex genome manipulation such as large deletion, addition, gene fusion and conditional removal of gene sequences at the target site. However, an efficient and easy-to-use Cre-recombinase delivery system remains lacking. Results: We designed and constructed two sets of expression vectors for Cre-recombinase using two highly efficient viral systems, the integrative lentivirus and non-integrative adeno associated virus...
2017: Journal of Biological Engineering
https://www.readbyqxmd.com/read/29203334/sex-differences-in-olfactory-induced-neural-activation-of-the-amygdala
#6
Takefumi Kikusui, Mayu Kajita, Natsumi Otsuka, Tatsuya Hattori, Kanako Kumazawa, Akiyuki Watarai, Miho Nagasawa, Ayumu Inutsuka, Akihiro Yamanaka, Naoki Matsuo, Herbert E Covington, Kazutaka Mogi
Olfactory signals, including the scent of urine, are thought to be processed by specific brain regions, such as the medial amygdala (Me), and regulate sexual behavior in a sex-dependent manner. We aimed to reveal the sex-specific neural circuit from the accessory olfactory bulb (AOB) to Me by using a transgenic mouse. We quantified the long-lasting green fluorescent protein (GFP) expression profile, which was controlled by the c-fos promotor in a sex-dependent manner by the scent of urine. Female urine predominantly activated neurons of the MePD in male mice and the MePV in female mice...
December 1, 2017: Behavioural Brain Research
https://www.readbyqxmd.com/read/29202463/patients-and-animal-models-of-cng%C3%AE-1-deficient-retinitis-pigmentosa-support-gene-augmentation-approach
#7
Simon M Petersen-Jones, Laurence M Occelli, Paige A Winkler, Winston Lee, Janet R Sparrow, Mai Tsukikawa, Sanford L Boye, Vince Chiodo, Jenina E Capasso, Elvir Becirovic, Christian Schön, Mathias W Seeliger, Alex V Levin, Stylianos Michalakis, William W Hauswirth, Stephen H Tsang
Retinitis pigmentosa (RP) is a major cause of blindness that affects 1.5 million people worldwide. Mutations in cyclic nucleotide-gated channel β 1 (CNGB1) cause approximately 4% of autosomal recessive RP. Gene augmentation therapy shows promise for treating inherited retinal degenerations; however, relevant animal models and biomarkers of progression in patients with RP are needed to assess therapeutic outcomes. Here, we evaluated RP patients with CNGB1 mutations for potential biomarkers of progression and compared human phenotypes with those of mouse and dog models of the disease...
November 20, 2017: Journal of Clinical Investigation
https://www.readbyqxmd.com/read/29201630/avoidance-of-harm-from-treatment-for-anca-associated-vasculitis
#8
REVIEW
Catherine King, Lorraine Harper
Purpose of review With established immunosuppressant treatment regimens for anti-neutrophil cytoplasm antibody-associated vasculitides (AAV), prognosis has significantly improved. The mainstay of treatment still comprises high-dose corticosteroids and cyclophosphamide for severe forms, although rituximab is being increasingly utilised instead of cyclophosphamide as induction therapy. AAV patients experience an excess of infections, malignancies and cardiovascular events as compared to the general population, which is a combination of the systemic inflammatory process associated with vasculitis and the adverse events from treatment...
2017: Current Treatment Options in Rheumatology
https://www.readbyqxmd.com/read/29199855/the-hierarchy-of-proinflammatory-cytokines-in-ocular-inflammation
#9
A P Da Cunha, Q Zhang, M Prentiss, X Q Wu, V Kainz, Y Y Xu, J Vrouvlianis, H Li, N Rangaswamy, B Leehy, T L McGee, C L Bell, C E Bigelow, V Kansara, Q Medley, Q Huang, H Y Wu
PURPOSE: The concept of tissue-dependent cytokine hierarchy has been demonstrated in a number of diseases, but it has not been investigated in ophthalmic diseases. Here, we evaluated the functional hierarchy of interleukin-1β (IL-1β), IL-6, IL-17A, and tumor necrosis factor (TNF) in the induction of ocular inflammation. MATERIALS AND METHODS: We delivered adeno-associated virus (AAV) vectors expressing IL-1β, IL-6, IL-17A, or TNF intravitreally in naïve C57/BL6 mice and compared and contrasted the inflammatory effects in the eye 5 weeks after AAV-mediated gene transfer...
December 4, 2017: Current Eye Research
https://www.readbyqxmd.com/read/29195742/preclinical-models-to-assess-the-immunogenicity-of-aav-vectors
#10
Hildegund C J Ertl
Although gene transfer using adeno-associated virus (AAV) vectors has made tremendous progress in recent years, challenges remain due to vector-specific adaptive immune responses. Specifically, AAV-neutralizing antibodies reduce AAV-transduction rates, while CD8+ T cells directed to AAV capsid antigens cause rejection of AAV-transduced cells. This has been addressed clinically by excluding humans with pre-existing AAV-neutralizing antibodies from gene transfer trials or by using immunosuppression or reduced doses of vectors expressing improved transgene products to blunt or circumvent destructive T cell responses...
November 23, 2017: Cellular Immunology
https://www.readbyqxmd.com/read/29192808/a-splice-site-variant-in-flvcr1-produces-retinitis-pigmentosa-without-posterior-column-ataxia
#11
Imran H Yusuf, Morag E Shanks, Penny Clouston, Robert E MacLaren
FLVCR1 (feline leukemia virus subgroup c receptor 1) is a transmembrane protein involved in the trafficking of intracellular heme. Homozygous variants in FLVCR1 have been described in association with a clinical syndrome of posterior column ataxia with retinitis pigmentosa (PCARP). Here, we describe a patient with non-syndromic retinitis pigmentosa homozygous for a splice-site variant in FLVCR1 (c.1092 + 5G>A) without evidence of posterior column ataxia or cerebellar degeneration. We suggest an association between intronic splice-site variants in FLVCR1 and the absence of posterior column degeneration and suggest a hypothesis to explain this observation...
December 1, 2017: Ophthalmic Genetics
https://www.readbyqxmd.com/read/29191939/effects-of-hypothalamic-leptin-gene-therapy-on-osteopetrosis-in-leptin-deficient-mice
#12
Kenneth A Philbrick, Stephen A Martin, Amy R Colagiovanni, Adam J Branscum, Russell T Turner, Urszula T Iwaniec
Impaired resorption of cartilage matrix deposited during endochondral ossification is a defining feature of juvenile osteopetrosis. Growing, leptin-deficient ob/ob mice exhibit a mild form of osteopetrosis. However, the extent to which the disease is (1) self-limiting and (2) reversible by leptin treatment is unknown. We addressed the first question by performing histomorphometric analysis of femurs in rapidly growing (2-month-old), slowly growing (4-month-old), and skeletally mature (6-month-old) wild type (WT) and ob/ob male mice...
November 30, 2017: Journal of Endocrinology
https://www.readbyqxmd.com/read/29191753/melanin-concentrating-hormone-acts-through-hypothalamic-kappa-opioid-system-and-p70s6k-to-stimulate-acute-food-intake
#13
Amparo Romero-Picó, Estrella Sanchez-Rebordelo, Monica Imbernon, David González-Touceda, Cintia Folgueira, Ana Senra, Johan Fernø, Clémence Blouet, Roberto Cabrera, Margriet van Gestel, Roger A Adan, Miguel López, Rafael Maldonado, Ruben Nogueiras, Carlos Diéguez
Melanin-Concentrating Hormone (MCH) is one of the most relevant orexigenic factors specifically located in the lateral hypothalamic area (LHA), with its physiological relevance demonstrated in studies using several genetically manipulated mice models. However, the central mechanisms controlling MCH-induced hyperphagia remain largely uncharacterized. Here, we show that central injection of MCH in mice deficient for kappa opoid receptor (k-OR) failed to stimulate feeding. To determine the hypothalamic area responsible for this MCH/k-OR interaction, we performed virogenetic studies and found that downregulation of k-OR by adeno-associated viruses (shOprk1-AAV) in LHA, but not in other hypothalamic nuclei, was sufficient to block MCH-induced food intake...
December 1, 2017: Neuropharmacology
https://www.readbyqxmd.com/read/29191327/optogenetic-modulation-of-cardiac%C3%A2-sympathetic-nerve-activity-to%C3%A2-prevent-ventricular%C3%A2-arrhythmias
#14
Lilei Yu, Liping Zhou, Gang Cao, Sunny S Po, Bing Huang, Xiaoya Zhou, Menglong Wang, Shenxu Yuan, Zhuo Wang, Songyun Wang, Hong Jiang
BACKGROUND: Studies have shown that left stellate ganglion (LSG) suppression protects against ventricular arrhythmias (VAs). Optogenetics is a novel technique to reversibly regulate the activity of the targeted neurons. OBJECTIVES: This study aimed to investigate whether an optogenetically silenced LSG could protect against VAs induced by myocardial ischemia. METHODS: Adeno-associated virus (AAV) was used as the vector to deliver ArchT, an inhibitory light-sensitive opsin, to the LSG neurons...
December 5, 2017: Journal of the American College of Cardiology
https://www.readbyqxmd.com/read/29188521/aav-serotype-testing-on-cultured-human-donor-retinal-explants
#15
Thilo M Buck, Lucie P Pellissier, Rogier M Vos, Elon H C van Dijk, Camiel J F Boon, Jan Wijnholds
This protocol details on a screening method for infectivity and tropism of different serotypes of adeno-associated viruses (AAVs) on human retinal explants with cell-type specific or ubiquitous green fluorescent protein (GFP) expression vectors. Eyes from deceased adult human donors are enucleated and the retinas are isolated. Each retina is punched into eight to ten 6-mm equal pieces. Whatman™ paper punches are placed on the retinas and the stack is transferred onto 24-well culture inserts with the photoreceptors facing the membrane...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29188520/production-of-ips-derived-human-retinal-organoids-for-use-in-transgene-expression-assays
#16
Peter M Quinn, Thilo M Buck, Charlotte Ohonin, Harald M M Mikkers, Jan Wijnholds
In vitro retinal organoid modeling from human pluripotent stem cells is becoming more common place in many ophthalmic laboratories worldwide. These organoids mimic human retinogenesis through formation of organized layered retinal structures that display markers for typical retinal cell types. Pivotally these humanized retinal models provide a stepping stone to the clinic as therapeutic tools and are expected to provide a promising alternative to current animal models. Thus pluripotent stem cell based healthy as well as diseased human retinal organoids are attractive for use in drug potency assays and gene augmentation therapeutics...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29188518/screening-for-neutralizing-antibodies-against-natural-and-engineered-aav-capsids-in-nonhuman-primate-retinas
#17
Timothy P Day, Leah C Byrne, John G Flannery, David V Schaffer
Adeno-associated virus (AAV) has shown promise as a therapeutic gene delivery vector for inherited retinal degenerations in both preclinical disease models and human clinical trials. The retinas of nonhuman primates (NHPs) share many anatomical similarities to humans and are an important model for evaluating AAV gene delivery. Recent evidence has shown that preexisting immunity in the form of neutralizing antibodies (NABs) in NHPs strongly correlates with weak or lack of AAV transduction in the retina when administered intravitreally, work with translational implications...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29188517/neutralizing-antibodies-against-adeno-associated-virus-aav-measurement-and-influence-on-retinal-gene-delivery
#18
Mélissa Desrosiers, Deniz Dalkara
Adeno-associated viral vectors have become widely used in the clinic for retinal gene therapy. Thanks to AAVs impeccable safety profile and positive functional outcomes in its clinical application, interest in retinal gene therapy has increased exponentially over the past decade. Although early clinical trials have shown there is little influence of neutralizing antibodies on the performance of AAV when vector is administered into the subretinal space, recent findings suggest neutralizing antibodies may play a role when AAV is delivered via the intravitreal route...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29188512/dual-aav-vectors-for-stargardt-disease
#19
Ivana Trapani
Stargardt disease (STGD1), due to mutations in the large ABCA4 gene, is the most common inherited macular degeneration in humans. Attempts at developing gene therapy approaches for treatment of STGD1 are currently ongoing. Among all the vectors available for gene therapy of inherited retinal diseases, those based on adeno-associated viruses (AAV) are the most promising given the efficacy shown in various animal models and their excellent safety profile in humans, as confirmed in many ongoing clinical trials...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/29188511/aav-gene-augmentation-therapy-for-crb1-associated-retinitis-pigmentosa
#20
C Henrique Alves, Jan Wijnholds
Mutations in the CRB1 gene account for around 10,000 persons with Leber congenital amaurosis (LCA) and 70,000 persons with retinitis pigmentosa (RP) worldwide. Therefore, the CRB1 gene is a key target in the fight against blindness. A proof-of-concept for an adeno-associated virus (AAV)-mediated CRB2 gene augmentation therapy for CRB1-RP was recently described. Preclinical studies using animal models such as knockout or mutant mice are crucial to obtain such proof-of-concept. In this chapter we describe a technique to deliver AAV vectors, into the murine retinas, via the subretinal route...
2018: Methods in Molecular Biology
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