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https://www.readbyqxmd.com/read/28225490/clinical-characteristics-and-outcome-of-spanish-patients-with-anca-associated-vasculitides-impact-of-the-vasculitis-type-anca-specificity-and-treatment-on-mortality-and-morbidity
#1
Roser Solans-Laqué, Guadalupe Fraile, Monica Rodriguez-Carballeira, Luis Caminal, Maria J Castillo, Ferran Martínez-Valle, Luis Sáez, Juan J Rios, Xavier Solanich, Joaquim Oristrell, Francisco Pasquau, Eva Fonseca, Monica Zamora, Jose L Callejas, Begoña Frutos, Monica Abdilla, Patricia Fanlo, Isabel García-Sánchez, Miguel López-Dupla, Bernardo Sopeña, Almudena Pérez-Iglesias, Josep A Bosch
The aim of this study was to describe the clinical characteristics of ANCA-associated vasculitides (AAV) at presentation, in a wide cohort of Spanish patients, and to analyze the impact of the vasculitis type, ANCA specificity, prognostic factors, and treatments administered at diagnosis, in the outcome.A total of 450 patients diagnosed between January 1990 and January 2014 in 20 Hospitals from Spain were included. Altogether, 40.9% had granulomatosis with polyangiitis (GPA), 37.1% microscopic polyangiitis (MPA), and 22% eosinophilic granulomatosis with polyangiitis (EGPA)...
February 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28221337/modulation-of-digital-flexor-tendon-healing-by-vascular-endothelial-growth-factor-gene-transfection-in-a-chicken-model
#2
W F Mao, Y F Wu, Q Q Yang, Y L Zhou, J B Tang
A major challenge in tendon injury is the weak intrinsic healing capacity of tendon, which may cause re-rupture after surgery. Growth factors are believed to be critical during tendon healing. This study aimed to investigate the effects of vascular endothelial growth factor (VEGF) genes delivered by adeno-associated virus (AAV) vectors on tendon healing and molecular events involved in a chicken model. One hundred and twenty eight deep flexor tendons in the long toes of chickens were completely transected and injected with 2 × 10(9) particles of AAV2-VEGF or saline before surgically repaired...
February 21, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28220790/in-vivo-genome-editing-with-a-small-cas9-orthologue-derived-from-campylobacter-jejuni
#3
Eunji Kim, Taeyoung Koo, Sung Wook Park, Daesik Kim, Kyoungmi Kim, Hee-Yeon Cho, Dong Woo Song, Kyu Jun Lee, Min Hee Jung, Seokjoong Kim, Jin Hyoung Kim, Jeong Hun Kim, Jin-Soo Kim
Several CRISPR-Cas9 orthologues have been used for genome editing. Here, we present the smallest Cas9 orthologue characterized to date, derived from Campylobacter jejuni (CjCas9), for efficient genome editing in vivo. After determining protospacer-adjacent motif (PAM) sequences and optimizing single-guide RNA (sgRNA) length, we package the CjCas9 gene, its sgRNA sequence, and a marker gene in an all-in-one adeno-associated virus (AAV) vector and produce the resulting virus at a high titer. CjCas9 is highly specific, cleaving only a limited number of sites in the human or mouse genome...
February 21, 2017: Nature Communications
https://www.readbyqxmd.com/read/28217942/hypertrophic-pachymeningitis-is-a-characteristic-manifestation-of-granulomatosis-with-polyangiitis-a-retrospective-study-of-anti-neutrophil-cytoplasmic-antibody-associated-vasculitis
#4
Yasuhiro Shimojima, Dai Kishida, Akiyo Hineno, Masahide Yazaki, Yoshiki Sekijima, Shu-Ichi Ikeda
AIM: To elucidate the characteristics of patients with hypertrophic pachymeningitis (HP) in a population with anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). METHODS: We retrospectively investigated the clinical records of 39 patients who were diagnosed with AAV. To determine the characteristics of HP in AAV, the epidemiological and clinical data from patients with HP were statistically compared with those from patients without HP. RESULTS: Of 39 patients with AAV, seven (17...
February 20, 2017: International Journal of Rheumatic Diseases
https://www.readbyqxmd.com/read/28212737/gene-delivery-of-apoptin-derived-peptide-using-an-adeno-associated-virus-vector-inhibits-glioma-and-prolongs-animal-survival
#5
Xiuli Zhong, Hengyu Zhao, Songhe Liang, DanYang Zhou, Wenjia Zhang, Lijie Yuan
Glioblastoma (GBM) is the most common malignant brain tumor in adults. We designed an adeno-associated virus (AAV) vector for intracranial delivery of the secreted HSP70-targeted peptide APOPTIN derived from Apoptin to GBM tumors. We applied this therapy to GBM models using human U87MG glioma cells and GBM xenograft models in mice. In U87MG and U251MG cells, conditioned medium from AAV2-apoptin-derived peptide (ADP)-expressing cells induced 83% and 78% cell death. In mice bearing intracranial U87MG tumors treated with AAV2-ADP, treatment resulted in a significant decrease in tumor growth and longer survival in mice bearing orthotopic invasive GBM brain tumors...
January 15, 2017: Biochemical and Biophysical Research Communications
https://www.readbyqxmd.com/read/28208742/aav-vector-mediated-gene-delivery-to-substantia-nigra-dopamine-neurons-implications-for-gene-therapy-and-disease-models
#6
REVIEW
Katrina Albert, Merja H Voutilainen, Andrii Domanskyi, Mikko Airavaara
Gene delivery using adeno-associated virus (AAV) vectors is a widely used method to transduce neurons in the brain, especially due to its safety, efficacy, and long-lasting expression. In addition, by varying AAV serotype, promotor, and titer, it is possible to affect the cell specificity of expression or the expression levels of the protein of interest. Dopamine neurons in the substantia nigra projecting to the striatum, comprising the nigrostriatal pathway, are involved in movement control and degenerate in Parkinson's disease...
February 8, 2017: Genes
https://www.readbyqxmd.com/read/28205311/management-of-cardiovascular-risk-factors-in-patients-with-anca-associated-vasculitis
#7
Carsten Paul Bramlage, Juliane Kröplin, Manuel Wallbach, Joan Minguet, Katherine Helen Smith, Stephan Lüders, Joachim Schrader, Susan Patschan, Oliver Gross, Cornelia Deutsch, Peter Bramlage, Gerhard Anton Müller, Michael Koziolek
OBJECTIVES: Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) is accompanied by increased cardiovascular (CV) risk. Treatment of AAV patients includes the management of conventional CV risk factors, primarily hypertension and hypercholesterolemia, while lipoprotein(a) (LP(a)) is an emerging potential target. METHODS: We performed a multicenter, retrospective study in Germany. Patients were considered if they were between 18 and 90 years old and presented with AAV...
February 16, 2017: Journal of Evaluation in Clinical Practice
https://www.readbyqxmd.com/read/28202741/seasonal-influence-on-the-risk-of-relapse-at-a-rise-of-antineutrophil-cytoplasmic-antibodies-in-vasculitis-patients-with-renal-involvement
#8
Michael J Kemna, Jan Willem Cohen Tervaert, Kelly Broen, Sjoerd A M E G Timmermans, Pieter van Paassen, Jan G M C Damoiseaux
OBJECTIVE: The objective of this study was to identify risk factors for a relapse at the time of an increase in antineutrophil cytoplasmic antibodies (ANCA) in patients with renal ANCA-associated vasculitis (AAV). METHODS: All patients between January 2000 and November 2011 with renal AAV having an ANCA rise during remission were included. Differences in time to relapse since the ANCA rise were assessed using a Cox regression model. The level of 25-hydroxy Vitamin D (25(OH)D) was assessed at the ANCA rise and at a subsequent relapse or time-matched during remission...
February 15, 2017: Journal of Rheumatology
https://www.readbyqxmd.com/read/28202570/aav-mediated-delivery-of-optogenetic-constructs-to-the-macaque-brain-triggers-humoral-immune-responses
#9
Skyler D Mendoza, Yasmine El-Shamayleh, Gregory D Horwitz
Gene delivery to the primate central nervous system via recombinant adeno-associated viral vectors (AAV) allows neurophysiologists to control and observe neural activity precisely. A current limitation of this approach is variability in vector transduction efficiency. Low levels of transduction can foil experimental manipulations, prompting vector readministration. The ability to make multiple vector injections into the same animal, even in cases where successful vector transduction has already been achieved, is also desirable...
February 15, 2017: Journal of Neurophysiology
https://www.readbyqxmd.com/read/28197125/rna-interference-of-human-%C3%AE-synuclein-in-mouse
#10
Young-Cho Kim, Adam Miller, Livia C R F Lins, Sang-Woo Han, Megan S Keiser, Ryan L Boudreau, Beverly L Davidson, Nandakumar S Narayanan
α-Synuclein is postulated to play a key role in the pathogenesis of Parkinson's disease (PD). Aggregates of α-synuclein contribute to neurodegeneration and cell death in humans and in mouse models of PD. Here, we use virally mediated RNA interference to knockdown human α-synuclein in mice. We used an siRNA design algorithm to identify eight siRNA sequences with minimal off-targeting potential. One RNA-interference sequence (miSyn4) showed maximal protein knockdown potential in vitro. We then designed AAV vectors expressing miSyn4 and injected them into the mouse substantia nigra...
2017: Frontiers in Neurology
https://www.readbyqxmd.com/read/28195574/muscle-specific-crispr-cas9-dystrophin-gene-editing-ameliorates-pathophysiology-in-a-mouse-model-for-duchenne-muscular-dystrophy
#11
Niclas E Bengtsson, John K Hall, Guy L Odom, Michael P Phelps, Colin R Andrus, R David Hawkins, Stephen D Hauschka, Joel R Chamberlain, Jeffrey S Chamberlain
Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. Here we develop multiple approaches for editing the mutation in dystrophic mdx(4cv) mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette together with single-guide RNA cassettes and, in one approach, a dystrophin homology region to fully correct the mutation...
February 14, 2017: Nature Communications
https://www.readbyqxmd.com/read/28195359/sodium-taurocholate-cotransporting-polypeptide-is-the-limiting-host-factor-of-hepatitis-b-virus-infection-in-macaque-and-pig-hepatocytes
#12
Florian A Lempp, Ellen Wiedtke, Bingqian Qu, Pierre Roques, Isabelle Chemin, Florian W R Vondran, Roger Le Grand, Dirk Grimm, Stephan Urban
: Infections with the human Hepatitis B (HBV) and Hepatitis D Virus (HDV) depend on species-specific host factors like the receptor human sodium taurocholate cotransporting polypeptide hNTCP. Complementation of mouse hepatocytes with hNTCP confers susceptibility to HDV but not HBV indicating the requirement of additional HBV-specific factors. As an essential premise towards the establishment for an HBV-susceptible animal model, we investigated the role of hNTCP as a limiting factor of hepatocytes in commonly used laboratory animals...
February 13, 2017: Hepatology: Official Journal of the American Association for the Study of Liver Diseases
https://www.readbyqxmd.com/read/28193235/dynamic-presenilin-1-and-synaptotagmin-1-interaction-modulates-exocytosis-and-amyloid-%C3%AE-production
#13
Katarzyna Marta Zoltowska, Masato Maesako, Iryna Lushnikova, Shuko Takeda, Laura J Keller, Galina Skibo, Bradley T Hyman, Oksana Berezovska
BACKGROUND: Alzheimer's disease (AD)-linked protein, presenilin 1 (PS1), is present at the synapse, and the knock-out of presenilin in mice leads to synaptic dysfunction. On the other hand, synaptic activity was shown to influence PS1-dependent generation of distinct amyloid β (Aβ) species. However, the precise nature of these regulations remains unclear. The current study reveals novel role of PS1 at the synapse, and deciphers how PS1 and synaptic vesicle-associated protein, synaptotagmin 1 (Syt1) modulate each other functions in neurons via direct activity-triggered interaction...
February 13, 2017: Molecular Neurodegeneration
https://www.readbyqxmd.com/read/28193101/how-to-successfully-screen-random-aav-display-peptide-libraries-in-vivo
#14
Jakob Körbelin, Martin Trepel
Adeno-associated virus (AAV) has emerged as a very promising gene therapy vector. To enable tissue-directed gene expression, many artificially generated AAV variants have been established, often isolated from large pools of mutated capsids. Random peptide libraries displayed on AAV capsids have been used successfully to select vectors targeted to a given target cell or tissue in vitro and in vivo. However, the published methodology for screening of AAV libraries to isolate vectors with selective tissue tropism after intravenous administration in vivo has not been described in sufficient detail to address all critical steps...
February 14, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28192678/assaying-the-stability-and-inactivation-of-aav-serotype-1-vectors
#15
Douglas B Howard, Brandon K Harvey
Adeno-associated virus (AAV) vectors are a commonplace tool for gene delivery ranging from cell culture to human gene therapy. One feature that makes AAV a desirable vector is its stability, in regard to both the duration of transgene expression and retention of infectivity as a viral particle. This study examined the stability of AAV serotype 1 (AAV1) vectors under different conditions. First, transducibility after storage at 4°C decreased 20% over 7 weeks. Over 10 freeze-thaw cycles, the resulting transduction efficiency became variable at 60-120% of a single thaw...
February 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28191609/complement-blockade-in-anca-associated-vasculitis-an-index-case-current-concepts-and-future-perspectives
#16
REVIEW
Lucio Manenti, Maria Letizia Urban, Federica Maritati, Maricla Galetti, Augusto Vaglio
Complement alternative pathway (cAP) hyperactivation seems to be involved in ANCA-associated vasculitis (AAV). We here describe a case of AAV with severe activation of cAP that developed acute renal failure. No mutation predisposing to cAP dysregulation was identified. We treated our patient with the standard immunosuppressive therapy, but disease progression was only reversed after the addition of eculizumab, a monoclonal antibody against C5; the patient eventually achieved an almost complete renal function recovery...
February 13, 2017: Internal and Emergency Medicine
https://www.readbyqxmd.com/read/28181422/hmgb1-contributes-to-glomerular-endothelial-cell-injury-in-anca-associated-vasculitis-through-enhancing-endothelium-neutrophil-interactions
#17
Chen Wang, Dong-Yuan Chang, Min Chen, Ming-Hui Zhao
Our previous studies demonstrated that high mobility group box-1 (HMGB1), a typical damage-associated molecular pattern (DAMP) protein, is associated with the disease activity of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV). Moreover, HMGB1 participates in ANCA-induced neutrophil activation. The current study aimed to investigate whether HMGB1 regulated the interaction between neutrophils and glomerular endothelial cells (GEnC) in the presence of ANCA. Correlation analysis on HMGB1 levels in AAV patients and soluble intercellular cell adhesion molecule-1 (sICAM-1) levels or vascular endothelial growth factor (VEGF) levels, which are markers of endothelial cell activation, was performed...
February 9, 2017: Journal of Cellular and Molecular Medicine
https://www.readbyqxmd.com/read/28179151/development-of-efficient-adeno-associated-virus-aav-mediated-gene-delivery-system-with-a-phytoactive-material-for-targeting-human-melanoma-cells
#18
John Hwan Lee, Yoojin Kim, Ye-Eun Yoon, Yong-Jin Kim, Seong-Geun Oh, Jae-Hyung Jang, Eunmi Kim
No abstract text is available yet for this article.
February 4, 2017: New Biotechnology
https://www.readbyqxmd.com/read/28177193/manufacturing-of-recombinant-adeno-associated-viruses-using-mammalian-expression-platforms
#19
REVIEW
Marc-André Robert, Parminder S Chahal, Alexandre Audy, Amine Kamen, Rénald Gilbert, Bruno Gaillet
Manufacturing practices for recombinant adeno-associated viruses (AAV) have improved in the last decade through the development of new platforms in conjunction with better production and purification methods. In this review, we discuss the advantages and limitations of the most popular systems and methods employed with mammalian cell platforms. Methods and systems such as transient transfection, packaging and producer cells and adenovirus and herpes simplex virus are described. In terms of best production yields, they are comparable with about 10(4) -10(5) vector genomes produced per cell but transient transfection of HEK293 cells is by far the most commonly used...
February 8, 2017: Biotechnology Journal
https://www.readbyqxmd.com/read/28169951/cardiac-gene-therapy-with-adeno-associated-virus-based-vectors
#20
Kyle Chamberlain, Jalish M Riyad, Thomas Weber
PURPOSE OF REVIEW: Cardiac gene therapy with adeno-associated virus (AAV)-based vectors is emerging as an entirely new platform to treat, or even cure, so far intractable cardiac disorders. This review describes our current knowledge of cardiac AAV gene therapy with a particular focus on the biggest obstacle for the successful translation of cardiac AAV gene therapy into the clinic, namely the efficient delivery of the therapeutic gene to the myocardium. RECENT FINDINGS: We summarize the significant recent progress that has been made in treating heart failure in preclinically relevant animal models with AAV gene therapy and the recent results of clinical trials with cardiac AAV gene therapy for the treatment of heart failure...
February 4, 2017: Current Opinion in Cardiology
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