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https://www.readbyqxmd.com/read/28648497/selective-optogenetic-control-of-purkinje-cells-in-monkey-cerebellum
#1
Yasmine El-Shamayleh, Yoshiko Kojima, Robijanto Soetedjo, Gregory D Horwitz
Purkinje cells of the primate cerebellum play critical but poorly understood roles in the execution of coordinated, accurate movements. Elucidating these roles has been hampered by a lack of techniques for manipulating spiking activity in these cells selectively-a problem common to most cell types in non-transgenic animals. To overcome this obstacle, we constructed AAV vectors carrying the channelrhodopsin-2 (ChR2) gene under the control of a 1 kb L7/Pcp2 promoter. We injected these vectors into the cerebellar cortex of rhesus macaques and tested vector efficacy in three ways...
June 21, 2017: Neuron
https://www.readbyqxmd.com/read/28648139/mutagenic-analysis-of-an-adeno-associated-virus-variant-capable-of-simultaneously-promoting-immune-resistance-and-robust-gene-delivery
#2
Yoojin Kim, Eunmi Kim, Seokmin Oh, Ye-Eun Yoon, Jae-Hyung Jang
In addition to the ability to boost gene delivery efficiency in many therapeutically relevant cells, the capability of circumventing neutralizing antibody (NAb) inactivation is a key prerequisite that gene carriers must fulfill for their extensive applications as therapeutic agents in many gene therapy trials, especially for cancer treatments. This study revealed that a genetically engineered adeno-associated viral (AAV) variant, AAVr3.45, inherently possesses dual beneficial properties as a gene carrier: i) efficiently delivering therapeutic genes to many clinically valuable cells (e...
June 24, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28646482/clinical-serological-and-histological-determinants-of-patient-and-renal-outcome-in-anca-associated-vasculitis-with-renal-involvement-an-analysis-from-a-referral-centre
#3
Matija Crnogorac, Ivica Horvatic, Luka Toric, Danica Galesic Ljubanovic, Miroslav Tisljar, Krešimir Galesic
PURPOSE: To evaluate significance of clinical and histopathological prognostic factors for renal and patient outcome in AAV patient cohort. METHODS: Retrospective study included consecutive patients diagnosed with pauci-immune crescentic glomerulonephritis from January 2003 to December 2013. Primary outcome was combined endpoint patient death or progression to end-stage renal disease (ESRD). Secondary outcomes were patient survival and progression to ESRD (renal survival) singularly and disease relapse...
June 23, 2017: International Urology and Nephrology
https://www.readbyqxmd.com/read/28646206/crispr-cas9-mediated-genome-editing-via-postnatal-administration-of-aav-vector-cures-haemophilia-b-mice
#4
Tsukasa Ohmori, Yasumitsu Nagao, Hiroaki Mizukami, Asuka Sakata, Shin-Ichi Muramatsu, Keiya Ozawa, Shin-Ichi Tominaga, Yutaka Hanazono, Satoshi Nishimura, Osamu Nureki, Yoichi Sakata
Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system. Administration of adeno-associated virus (AAV) 8 vector harbouring Staphylococcus aureus Cas9 (SaCas9) and single guide RNA (sgRNA) to wild-type adult mice induced a double-strand break (DSB) at the target site of F9 in hepatocytes, sufficiently developing haemophilia B...
June 23, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28645365/complexity-of-immune-responses-to-aav-transgene-products-example-of-factor-ix
#5
REVIEW
Roland W Herzog
After two decades of research, in vivo gene transfer with adeno-associated viral (AAV) vectors has now resulted in successful treatments and even cures for several human diseases. However, the potential for immune responses against the therapeutic gene products remains one of the concerns as this approach is broadened to more patients, diverse diseases, and target organs. Immune responses following gene transfer of coagulation factor IX (FIX) for the treatment of the bleeding disorder hemophilia B has been extensively investigated in multiple animal models...
May 29, 2017: Cellular Immunology
https://www.readbyqxmd.com/read/28645308/neonatal-aav-delivery-of-alpha-synuclein-induces-pathology-in-the-adult-mouse-brain
#6
Marion Delenclos, Ayman H Faroqi, Mei Yue, Aishe Kurti, Monica Castanedes-Casey, Linda Rousseau, Virginia Phillips, Dennis W Dickson, John D Fryer, Pamela J McLean
Abnormal accumulation of alpha-synuclein (αsyn) is a pathological hallmark of Lewy body related disorders such as Parkinson's disease and Dementia with Lewy body disease. During the past two decades, a myriad of animal models have been developed to mimic pathological features of synucleinopathies by over-expressing human αsyn. Although different strategies have been used, most models have little or no reliable and predictive phenotype. Novel animal models are a valuable tool for understanding neuronal pathology and to facilitate development of new therapeutics for these diseases...
June 23, 2017: Acta Neuropathologica Communications
https://www.readbyqxmd.com/read/28642036/transcription-factor-decoy-technology-a-therapeutic-update
#7
REVIEW
Markus Hecker, Andreas H Wagner
Targeting transcription factors represents one possibility to interfere with a known activated regulatory pathway that promotes disease. Double-stranded transcription factor decoy (TFD) oligodeoxynucleotides (ODN) are therapeutic drug candidates, which are able to specifically target and neutralize key transcription factors involved in the pathogenesis of a given disease. These short double-stranded TFD molecules mimic the consensus DNA binding site of a specific transcription factor in the promoter region of its target genes...
June 19, 2017: Biochemical Pharmacology
https://www.readbyqxmd.com/read/28638915/-optogenetic-activation-of-dorsal-hippocampal-astrocytic-rac1-blocks-the-learning-of-associative-memory
#8
Xiao-Mu Guo, Zhao-Hui Liao, Ye-Zheng Tao, Fei-Fei Wang, Lan Ma
Rac1 belongs to the family of Rho GTPases, and plays important roles in the brain function. It affects the cell migration and axon guidance via regulating the cytoskeleton and cellular morphology. However, the effect of its dynamic activation in regulating physiological function remains unclear. Recently, a photoactivatable analogue of Rac1 (PA-Rac1) has been developed, allowing the activation of Rac1 by the specific wavelength of light in living cells. Thus, we constructed recombinant adeno-associated virus (AAV) of PA-Rac1 and its light-insensitive mutant PA-Rac1-C450A under the control of the mouse glial fibrillary acidic protein (mGFAP) promoter to manipulate Rac1 activity in astrocytes by optical stimulation...
June 25, 2017: Sheng Li Xue Bao: [Acta Physiologica Sinica]
https://www.readbyqxmd.com/read/28634402/enhanced-antiviral-and-antifibrotic-effects-of-short-hairpin-rnas-targeting-hbv-and-tgf-%C3%AE-in-hbv-persistent-mice
#9
Lei Ye, Fangming Kan, Tao Yan, Jiaqi Cao, Leiliang Zhang, Zhijian Wu, Wuping Li
The hepatitis B virus (HBV) causes acute and chronic liver infection, which may lead to liver cirrhosis and hepatocellular carcinoma. Current treatments including interferons and nucleotide analogs, have limited therapeutic effects, underscoring the need to identify effective therapeutic options to inhibit HBV replication and prevent complications. Previous animal models mimicking chronic HBV infection do not faithfully reflect disease progression in humans. Here, we used our established HBV-persistent mouse line with liver fibrosis to evaluate the efficacy of novel therapies...
June 20, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28629856/ethnic-differences-in-glucose-effectiveness-and-disposition-index-in-overweight-obese-african-american-and-white-women-with-prediabetes-a-study-of-compensatory-mechanisms
#10
Kwame Osei, Trudy Gaillard
OBJECTIVE: Prediabetes, a major precursor of type 2 diabetes, varies among ethnic populations. Therefore, we compared the pathophysiologic mechanisms of prediabetes in overweight/obese African American (AA) and White American (WA) women. SUBJECTS AND METHODS: We recruited 95 women (67 AA, 28 WA) with prediabetes. Standard OGTT and FSIVGTT were performed in each subject. Insulin sensitivity (Si), glucose effectiveness (Sg), beta cell function (acute insulin response to glucose (AIRg) and disposition index (DI: Si×AIRg) were calculated using Bergman's Minmod...
March 14, 2017: Diabetes Research and Clinical Practice
https://www.readbyqxmd.com/read/28629822/structure-based-designed-nano-dysferlin-significantly-improves-dysferlinopathy-in-bla-j-mice
#11
Telmo Llanga, Nadia Nagy, Laura Conatser, Catherine Dial, R Bryan Sutton, Matthew L Hirsch
Dysferlinopathy is an autosomal recessive muscular dystrophy characterized by the progressive loss of motility that is caused by mutations throughout the DYSF gene. There are currently no approved therapies that ameliorate or reverse dysferlinopathy. Gene delivery using adeno-associated vectors (AAVs) is a leading therapeutic strategy for genetic diseases; however, the large size of dysferlin cDNA (6.2 kB) precludes packaging into a single AAV capsid. Therefore, using 3D structural modeling and hypothesizing dysferlin C2 domain redundancy, a 30% smaller, dysferlin-like molecule amenable to single AAV vector packaging was engineered (termed Nano-Dysferlin)...
June 16, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28629744/mannose-receptor-modulates-macrophage-polarization-and-allergic-inflammation-through-mir-511-3p
#12
Yufeng Zhou, Danh C Do, Faoud T Ishmael, Mario Leonardo Squadrito, Ho Man Tang, Ho Lam Tang, Man-Hsun Hsu, Lipeng Qiu, Changjun Li, Yongqing Zhang, Kevin G Becker, Mei Wan, Shau-Ku Huang, Peisong Gao
BACKGROUND: Mannose receptor (MRC1/CD206) has been suggested to mediate allergic sensitization and asthma to multiple glyco-allergens, including cockroach allergens. OBJECTIVE: Determine the existence of a protective mechanism through which MRC1 limits allergic inflammation through its intronic miR-511-3p. METHODS: We examined the MRC1-mediated cockroach allergen uptake by lung macrophages and lung inflammation using C57BL/6 wild-type (WT) and Mrc1(-/-) mice...
June 16, 2017: Journal of Allergy and Clinical Immunology
https://www.readbyqxmd.com/read/28628231/gene-therapy-decreases-seizures-in-a-model-of-incontinentia-pigmenti
#13
Godwin K Dogbevia, Kathrin Töllner, Jakob Körbelin, Sonja Bröer, Dirk A Ridder, Hanna Grasshoff, Claudia Brandt, Jan Wenzel, Beate K Straub, Martin Trepel, Wolfgang Löscher, Markus Schwaninger
OBJECTIVE: Incontinentia pigmenti (IP) is a genetic disease leading to severe neurological symptoms, such as epileptic seizures, but no specific treatment is available. IP is caused by pathogenic variants that inactivate the Nemo gene. Replacing Nemo through gene therapy might provide therapeutic benefits. METHODS: In a mouse model of IP we administered a single intravenous dose of the AAV vector AAV-BR1-CAG-NEMO delivering the Nemo gene to the brain endothelium...
June 19, 2017: Annals of Neurology
https://www.readbyqxmd.com/read/28628105/identification-of-liver-specific-enhancer-promoter-activity-in-the-3-untranslated-region-of-the-wild-type-aav2-genome
#14
Grant J Logan, Allison P Dane, Claus V Hallwirth, Christine M Smyth, Emilie E Wilkie, Anais K Amaya, Erhua Zhu, Neeta Khandekar, Samantha L Ginn, Sophia H Y Liao, Sharon C Cunningham, Natsuki Sasaki, Martí Cabanes-Creus, Patrick P L Tam, David W Russell, Leszek Lisowski, Ian E Alexander
Vectors based on adeno-associated virus type 2 (AAV2) are powerful tools for gene transfer and genome editing applications. The level of interest in this system has recently surged in response to reports of therapeutic efficacy in human clinical trials, most notably for those in patients with hemophilia B (ref. 3). Understandably, a recent report drawing an association between AAV2 integration events and human hepatocellular carcinoma (HCC) has generated controversy about the causal or incidental nature of this association and the implications for AAV vector safety...
June 19, 2017: Nature Genetics
https://www.readbyqxmd.com/read/28627251/direct-lc-ms-analysis-for-complete-characterization-of-recombinant-adeno-associated-virus-capsid-proteins
#15
Xiaoying Jin, Lin Liu, Shelley Nass, Catherine R O'Riordan, Eric Pastor, Kate Xiaokui Zhang
The requirement for robust analytical methods to support characterization of Adeno-Associated Virus (AAV) vectors is immediate, as the field advances more AAV gene therapeutics into the clinic and onto commercialization. AAV capsid proteins (VPs) are critical for viral infectivity and vector potency, thus, complete characterization of the constituent viral capsid proteins of AAV vector therapeutics, including their sequence and post-translational modifications (PTMs), is highly recommended to ensure AAV product quality and consistency...
June 18, 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28626777/long-term-efficacy-and-safety-of-insulin-and-glucokinase-gene-therapy-for-diabetes-8-year-follow-up-in-dogs
#16
Maria Luisa Jaén, Laia Vilà, Ivet Elias, Veronica Jimenez, Jordi Rodó, Luca Maggioni, Rafael Ruiz-de Gopegui, Miguel Garcia, Sergio Muñoz, David Callejas, Eduard Ayuso, Tura Ferré, Iris Grifoll, Anna Andaluz, Jesus Ruberte, Virginia Haurigot, Fatima Bosch
Diabetes is a complex metabolic disease that exposes patients to the deleterious effects of hyperglycemia on various organs. Achievement of normoglycemia with exogenous insulin treatment requires the use of high doses of hormone, which increases the risk of life-threatening hypoglycemic episodes. We developed a gene therapy approach to control diabetic hyperglycemia based on co-expression of the insulin and glucokinase genes in skeletal muscle. Previous studies proved the feasibility of gene delivery to large diabetic animals with adeno-associated viral (AAV) vectors...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28624194/sustained-inhibition-of-hbv-replication-in%C3%A2-vivo-after-systemic-injection-of-aavs-encoding-artificial-antiviral-primary-micrornas
#17
Mohube Betty Maepa, Abdullah Ely, Wayne Grayson, Patrick Arbuthnot
Chronic infection with hepatitis B virus (HBV) remains a problem of global significance and improving available treatment is important to prevent life-threatening complications arising in persistently infected individuals. HBV is susceptible to silencing by exogenous artificial intermediates of the RNA interference (RNAi) pathway. However, toxicity of Pol III cassettes and short duration of silencing by effectors of the RNAi pathway may limit anti-HBV therapeutic utility. To advance RNAi-based HBV gene silencing, mono- and trimeric artificial primary microRNAs (pri-miRs) derived from pri-miR-31 were placed under control of the liver-specific modified murine transthyretin promoter...
June 16, 2017: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/28623488/vasculitis-for-the-internist-focus-on-anca-associated-vasculitis
#18
REVIEW
Benjamin Chaigne, Loïc Guillevin
Vasculitides comprise several diseases affecting vessels of different sizes, mainly arteries, but also capillaries and veins. Antineutrophil cytoplasm antibody-associated vasculitides (AAVs) belong to the systemic necrotizing small-vessel vasculitis group that comprises granulomatosis with polyangiitis, microscopic polyangiitis and eosinophilic granulomatosis with polyangiitis. Internists often see AAV patients, and although their outcomes are usually good, the disease can be responsible for morbidity and mortality...
June 16, 2017: Internal and Emergency Medicine
https://www.readbyqxmd.com/read/28622288/optimization-of-design-and-production-strategies-for-novel-adeno-associated-viral-display-peptide-libraries
#19
J Körbelin, A Hunger, M Alawi, T Sieber, M Binder, M Trepel
Libraries displaying random peptides on the surface of adeno-associated virus (AAV) are powerful tools for the generation of target-specific gene therapy vectors. However, for unknown reasons the success rate of AAV library screenings is variable and the influence of the production procedure has not been thoroughly evaluated. During library screenings, the capsid variants with the most favorable tropism are enriched over several selection rounds on a target of choice and identified by subsequent sequencing of the encapsidated viral genomes encoding the library capsids with targeting peptide insertions...
June 16, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28619113/mechanisms-underlying-extensive-ser129-phosphorylation-in-%C3%AE-synuclein-aggregates
#20
Shigeki Arawaka, Hiroyasu Sato, Asuka Sasaki, Shingo Koyama, Takeo Kato
Parkinson's disease (PD) is characterized neuropathologically by intracellular aggregates of fibrillar α-synuclein, termed Lewy bodies (LBs). Approximately 90% of α-synuclein deposited as LBs is phosphorylated at Ser129 in brains with PD. In contrast, only 4% of total α-synuclein is phosphorylated at Ser129 in brains with normal individuals. It is unclear why extensive phosphorylation occurs in the pathological process of PD. To address this issue, we investigated a mechanism and role of Ser129-phosphorylation in regulating accumulation of α-synuclein...
June 15, 2017: Acta Neuropathologica Communications
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