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adeno-associated viral

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https://www.readbyqxmd.com/read/28433936/sustained-viral-gene-delivery-from-a-micro-fibrous-elastomeric-cardiac-patch-to-the-ischemic-rat-heart
#1
Xinzhu Gu, Yasumoto Matsumura, Ying Tang, Souvik Roy, Richard Hoff, Bing Wang, William R Wagner
Biodegradable and elastomeric patches have been applied to the surface of infarcted hearts as temporary mechanical supports to effectively alter adverse left ventricular remodeling processes. In this report, recombinant adeno-associated virus (AAV), known for its persistent transgene expression and low pathogenicity, was incorporated into elastomeric polyester urethane urea (PEUU) and polyester ether urethane urea (PEEUU) and processed by electrospinning into two formats (solid fibers and core-sheath fibers) designed to influence the controlled release behavior...
April 14, 2017: Biomaterials
https://www.readbyqxmd.com/read/28432975/vaccination-strategies-against-zika-virus
#2
REVIEW
Estefania Fernandez, Michael S Diamond
The epidemic emergence of Zika virus (ZIKV) in 2015-2016 has been associated with congenital malformations and neurological sequela. Current efforts to develop a ZIKV vaccine build on technologies that successfully reduced infection or disease burden against closely related flaviviruses or other RNA viruses. Subunit-based (DNA plasmid and modified mRNA), viral vectored (adeno- and measles viruses) and inactivated viral vaccines are already advancing to clinical trials in humans after successful mouse and non-human primate studies...
April 19, 2017: Current Opinion in Virology
https://www.readbyqxmd.com/read/28424574/present-molecular-limitations-of-on-bipolar-cell-targeted-gene-therapy
#3
Michiel van Wyk, Elmar C Hulliger, Lara Girod, Andreas Ebneter, Sonja Kleinlogel
Recent studies have demonstrated the safety and efficacy of ocular gene therapy based on adeno-associated viral vectors (AAVs). Accordingly, a surge in promising new gene therapies is entering clinical trials, including the first optogenetic therapy for vision restoration. To date, optogenetic therapies for vision restoration target either the retinal ganglion cells (GCs) or presynaptic ON-bipolar cells (OBCs). Initiating light responses at the level of the OBCs has significant advantages over optogenetic activation of GCs...
2017: Frontiers in Neuroscience
https://www.readbyqxmd.com/read/28423326/rapid-molecular-profiling-of-defined-cell-types-using-viral-trap
#4
Alexander R Nectow, Maria V Moya, Mats I Ekstrand, Awni Mousa, Kelly L McGuire, Caroline E Sferrazza, Bianca C Field, Gabrielle S Rabinowitz, Kirsty Sawicka, Yupu Liang, Jeffrey M Friedman, Nathaniel Heintz, Eric F Schmidt
Translational profiling methodologies enable the systematic characterization of cell types in complex tissues, such as the mammalian brain, where neuronal isolation is exceptionally difficult. Here, we report a versatile strategy for profiling CNS cell types in a spatiotemporally restricted fashion by engineering a Cre-dependent adeno-associated virus expressing an EGFP-tagged ribosomal protein (AAV-FLEX-EGFPL10a) to access translating mRNAs by translating ribosome affinity purification (TRAP). We demonstrate the utility of this AAV to target a variety of genetically and anatomically defined neural populations expressing Cre recombinase and illustrate the ability of this viral TRAP (vTRAP) approach to recapitulate the molecular profiles obtained by bacTRAP in corticothalamic neurons across multiple serotypes...
April 18, 2017: Cell Reports
https://www.readbyqxmd.com/read/28420695/adeno-associated-viral-9-mediated-cdk5-inhibitory-peptide-reverses-pathologic-changes-and-behavioral-deficits-in-the-alzheimer-s-disease-mouse-model
#5
Yong He, Suyue Pan, Miaojing Xu, Rongni He, Wei Huang, Pingping Song, Jianou Huang, Han-Ting Zhang, Yafang Hu
Cyclin-dependent kinase 5 (Cdk5), which binds to and is activated by p35, phosphorylates multiple substrates and plays an essential role in the development and function of the CNS; however, proteolytic production of p25 from p35 under stress conditions leads to the inappropriate activation of Cdk5 and contributes to hyperphosphorylation of τ and other substrates that are related to the pathogenesis of Alzheimer's disease. Selective inhibition of aberrant Cdk5 activity via genetic overexpression of Cdk5 inhibitory peptide (CIP) reduces pathologic changes and prevents brain atrophy and memory loss in p25-transgenic mice...
April 18, 2017: FASEB Journal: Official Publication of the Federation of American Societies for Experimental Biology
https://www.readbyqxmd.com/read/28417953/recombinant-adeno-associated-virus-mediated-rescue-of-function-in-a-mouse-model-of-dopamine-transporter-deficiency-syndrome
#6
P Illiano, C E Bass, L Fichera, L Mus, E A Budygin, T D Sotnikova, D Leo, S Espinoza, R R Gainetdinov
Dopamine Transporter Deficiency Syndrome (DTDS) is a rare autosomal recessive disorder caused by loss-of-function mutations in dopamine transporter (DAT) gene, leading to severe neurological disabilities in children and adults. DAT-Knockout (DAT-KO) mouse is currently the best animal model for this syndrome, displaying functional hyperdopaminergia and neurodegenerative phenotype leading to premature death in ~36% of the population. We used DAT-KO mouse as model for DTDS to explore the potential utility of a novel combinatorial adeno-associated viral (AAV) gene therapy by expressing DAT selectively in DA neurons and terminals, resulting in the rescue of aberrant striatal DA dynamics, reversal of characteristic phenotypic and behavioral abnormalities, and prevention of premature death...
April 18, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28411016/gene-therapy-for-hemophilia
#7
REVIEW
Arthur W Nienhuis, Amit C Nathwani, Andrew M Davidoff
The X-linked bleeding disorder hemophilia causes frequent and exaggerated bleeding that can be life-threatening if untreated. Conventional therapy requires frequent intravenous infusions of the missing coagulation protein (factor VIII [FVIII] for hemophilia A and factor IX [FIX] for hemophilia B). However, a lasting cure through gene therapy has long been sought. After a series of successes in small and large animal models, this goal has finally been achieved in humans by in vivo gene transfer to the liver using adeno-associated viral (AAV) vectors...
April 11, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28409504/comparative-study-of-the-transfection-efficiency-of-commonly-used-viral-vectors-in-rhesus-monkey-macaca-mulatta-brains
#8
Shi-Hao Wu, Zhi-Xing Liao, Joshua D Rizak, Na Zheng, Lin-Heng Zhang, Hen Tang, Xiao-Bin He, Yang Wu, Xia-Ping He, Mei-Feng Yang, Zheng-Hui Li, Dong-Dong Qin, Xin-Tian Hu
Viral vector transfection systems are among the simplest of biological agents with the ability to transfer genes into the central nervous system. In brain research, a series of powerful and novel gene editing technologies are based on these systems. Although many viral vectors are used in rodents, their full application has been limited in non-human primates. To identify viral vectors that can stably and effectively express exogenous genes within non-human primates, eleven commonly used recombinant adeno-associated viral and lentiviral vectors, each carrying a gene to express green or red fluorescence, were injected into the parietal cortex of four rhesus monkeys...
March 18, 2017: Zoological Research
https://www.readbyqxmd.com/read/28398005/nanotherapy-for-duchenne-muscular-dystrophy
#9
REVIEW
Michael E Nance, Chady H Hakim, N Nora Yang, Dongsheng Duan
Duchenne muscular dystrophy (DMD) is a lethal X-linked childhood muscle wasting disease caused by mutations in the dystrophin gene. Nanobiotechnology-based therapies (such as synthetic nanoparticles and naturally existing viral and nonviral nanoparticles) hold great promise to replace and repair the mutated dystrophin gene and significantly change the disease course. While a majority of DMD nanotherapies are still in early preclinical development, several [such as adeno-associated virus (AAV)-mediated systemic micro-dystrophin gene therapy] are advancing for phase I clinical trials...
April 11, 2017: Wiley Interdisciplinary Reviews. Nanomedicine and Nanobiotechnology
https://www.readbyqxmd.com/read/28393043/leber-s-congenital-amaurosis-and-the-role-of-gene-therapy-in-congenital-retinal-disorders
#10
REVIEW
Walid Sharif, Zuhair Sharif
Leber's congenital amaurosis (LCA) and recent gene therapy advancement for treating inherited retinopathies were extensive literature reviewed using MEDLINE, PubMed and EMBASE. Adeno-associated viral vectors were the most utilised vectors for ocular gene therapy. Cone photoreceptor cells might use an alternate pathway which was not reliant of the retinal pigment epithelium (RPE) derived retinoid isomerohydrolase (RPE65) to access the 11-cis retinal dehydechromophore. Research efforts dedicated on the progression of a gene-based therapy for the treatment of LCA2...
2017: International Journal of Ophthalmology
https://www.readbyqxmd.com/read/28387350/recombinant-adeno-associated-virus-mediated-expression-of-methamphetamine-antibody-attenuates-methamphetamine-induced-hyperactivity-in-mice
#11
Yun-Hsiang Chen, Kuo-Jen Wu, Kuang-Lun Wu, Kun-Lieh Wu, Ho-Min Tsai, Mao-Liang Chen, Yi-Wei Chen, Wei Hsieh, Chun-Ming Lin, Yun Wang
Methamphetamine (Meth) is one of the most frequently abused drugs worldwide. Recent studies have indicated that antibodies with high affinity for Meth reduce its pharmacological effects. The purpose of this study was to develop a technique for virus-based passive immunization against Meth effects. We generated a recombinant adeno-associated virus serotype-8 vector (AAV-MethAb) carrying the gene for a Meth-specific monoclonal antibody (MethAb). Infection of 293 cells with AAV-MethAb resulted in the expression and secretion of antibodies which bind to Meth...
April 7, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28372296/new-cell-engineering-approaches-for-cartilage-regenerative-medicine
#12
Magali Cucchiarini
Articular cartilage injuries have an inadequate aptitude to reproduce the original structure and functions of this highly specialized tissue. As most of the currently available options also do not lead to the restoration of the original hyaline cartilage, novel treatments are critically needed to address this global problems in the clinics. Gene therapy combined with tissue engineering approaches offers effective tools capable of enhancing cartilage repair experimentally, especially those based on the controlled delivery of the highly effective, clinically adapted recombinant adeno-associated viral (rAAV) vectors...
2017: Bio-medical Materials and Engineering
https://www.readbyqxmd.com/read/28370029/long-term-effects-of-systemic-gene-therapy-in-a-canine-model-of-myotubular-myopathy
#13
Matthew Elverman, Melissa A Goddard, David Mack, Jessica M Snyder, Michael W Lawlor, Hui Meng, Alan H Beggs, Ana Buj-Bello, Karine Poulard, Anthony P Marsh, Robert W Grange, Valerie E Kelly, Martin K Childers
INTRODUCTION: X-linked myotubular myopathy (XLMTM), a devastating pediatric disease caused by the absence of the protein myotubularin, results from mutations in the MTM1 gene. While there is no cure for XLMTM, we previously reported effects of MTM1 gene therapy using adeno-associated viral (AAV) vector on muscle weakness and pathology in MTM1-mutant dogs. Here, we followed 2 AAV-infused dogs over 4 years. METHODS: We evaluated gait, strength, respiration, neurological function, muscle pathology, AAV vector copy number (VCN), and transgene expression...
March 29, 2017: Muscle & Nerve
https://www.readbyqxmd.com/read/28367882/severe-acute-respiratory-infection-sari-surveillance-for-hospitalized-patients-in-northern-vietnam-2011-2014
#14
Hang Khanh Le Nguyen, Son Vu Nguyen, Anh Phuong Nguyen, Phuong Mai Vu Hoang, Thanh Thi Le, Thach Co Nguyen, Huong Thu Hoang, Cuong Duc Vuong, Loan Thi Thanh Tran, Le Quynh Mai
Worldwide Severe Acute Respiratory Infections (SARI) are leading causes of hospitalization, morbidity and mortality in children. The aim of this study was to identify viral pathogens responsible for SARI in northern Vietnam from 2011 to 2014. METHODS: Throat swabs and tracheal aspirates were collected from SARI patients according to WHO Guidelines. Thirteen different viral pathogens (Influenza A(H1N)pdm09; A/H3N2; A/H5; A/H7 and B; Para influenza 1,2,3; RSV; HMPV; Adeno; SARS-CoV and Rhino) were tested by conventional/ real-time RT-PCR...
March 28, 2017: Japanese Journal of Infectious Diseases
https://www.readbyqxmd.com/read/28366764/in%C3%A2-vivo-excision-of-hiv-1-provirus-by-sacas9-and-multiplex-single-guide-rnas-in-animal-models
#15
Chaoran Yin, Ting Zhang, Xiying Qu, Yonggang Zhang, Raj Putatunda, Xiao Xiao, Fang Li, Weidong Xiao, Huaqing Zhao, Shen Dai, Xuebin Qin, Xianming Mo, Won-Bin Young, Kamel Khalili, Wenhui Hu
CRISPR-associated protein 9 (Cas9)-mediated genome editing provides a promising cure for HIV-1/AIDS; however, gene delivery efficiency in vivo remains an obstacle to overcome. Here, we demonstrate the feasibility and efficiency of excising the HIV-1 provirus in three different animal models using an all-in-one adeno-associated virus (AAV) vector to deliver multiplex single-guide RNAs (sgRNAs) plus Staphylococcus aureus Cas9 (saCas9). The quadruplex sgRNAs/saCas9 vector outperformed the duplex vector in excising the integrated HIV-1 genome in cultured neural stem/progenitor cells from HIV-1 Tg26 transgenic mice...
March 30, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28355224/generation-of-infectious-recombinant-adeno-associated-virus-in-saccharomyces-cerevisiae
#16
Daniel Barajas, Juan Jose Aponte-Ubillus, Hassibullah Akeefe, Tomas Cinek, Joseph Peltier, Daniel Gold
The yeast Saccharomyces cerevisiae has been successfully employed to establish model systems for a number of viruses. Such model systems are powerful tools to study the virus biology and in particular for the identification and characterization of host factors playing a role in the viral infection cycle. Adeno-associated viruses (AAV) are heavily studied due to their use as gene delivery vectors. AAV relies on other helper viruses for successful replication and on host factors for several aspects of the viral life cycle...
2017: PloS One
https://www.readbyqxmd.com/read/28348718/intramyocardial-injection-of-recombinant-adeno-associated-viral-vector-coexpressing-pr39-adrenomedullin-enhances-angiogenesis-and-reduces-apoptosis-in-a-rat-myocardial-infarction-model
#17
Rui An, Cong Xi, Jian Xu, Ying Liu, Shumiao Zhang, Yuemin Wang, Yuewen Hao, Lijun Sun
Cotransfer of angiogenic and antiapoptotic genes could be the basis of new gene therapy strategies for myocardial infarction. In this study, rAAV-PR39-ADM, coexpressing antimicrobial peptide (PR39) and adrenomedullin (ADM), was designed with the mediation of recombinant adeno-associated virus. In vitro, CRL-1730 cells were divided into four groups, namely, the sham group, the AAV-null group, the NS (normal saline) group, and the PR39-ADM group. Immunocytochemistry analysis, CCK-8 assays, Matrigel assays, and apoptotic analysis were performed; in vivo, myocardial infarction model was established through ligation of the left coronary artery on rats, and treatment groups corresponded to those used in vitro...
2017: Oxidative Medicine and Cellular Longevity
https://www.readbyqxmd.com/read/28345428/induction-of-t-cell-infiltration-and-programmed-death-ligand-2-expression-by-adeno-associated-virus-in-rhesus-macaque-skeletal-muscle-and-modulation-by-prednisone
#18
Megan L Cramer, Guohong Shao, Louise R Rodino-Klapac, Louis G Chicoine, Paul T Martin
Use of adeno-associated virus (AAV) to transduce genes into skeletal muscles can be associated with T-cell responses to viral capsid and/or to transgenic protein. Intramuscular mononuclear cell infiltrates primarily consisting of CD8+ T cells and also containing FOXP3+ regulatory T cells were present in rhesus macaque skeletal muscle treated with rAAVrh74.MCK.GALGT2 by vascular delivery. Administration of oral prednisone prior to AAV gene delivery and throughout the study reduced such infiltrates by 60% at 24 weeks post AAV delivery compared with AAV-treated animals not receiving prednisone, regardless of the presence of pre-existing AAV serum antibodies at the time of treatment...
March 23, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28344998/low-dose-liver-targeted-gene-therapy-for-pompe-disease-enhances-therapeutic-efficacy-of-ert-via-immune-tolerance-induction
#19
Sang-Oh Han, Giuseppe Ronzitti, Benjamin Arnson, Christian Leborgne, Songtao Li, Federico Mingozzi, Dwight Koeberl
Pompe disease results from acid α-glucosidase (GAA) deficiency, and enzyme replacement therapy (ERT) with recombinant human (rh) GAA has clinical benefits, although its limitations include the short half-life of GAA and the formation of antibody responses. The present study compared the efficacy of ERT against gene transfer with an adeno-associated viral (AAV) vector containing a liver-specific promoter. GAA knockout (KO) mice were administered either a weekly injection of rhGAA (20 mg/kg) or a single injection of AAV2/8-LSPhGAA (8 × 10(11) vector genomes [vg]/kg)...
March 17, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28339926/study-on-the-expression-of-human-lysozyme-in-oviduct-bioreactor-mediated-by-recombinant-avian-adeno-associated-virus1
#20
A P Wang, Y J Wang, S Wu, W Y Zuo, C M Guo, W M Hong, S Y Zhu
Due to its antimicrobial properties and low toxicity, human lysozyme (hLYZ) has broad application in the medical field and as a preservative used by the food industry. However, limited availability hinders its widespread use. Hence, we constructed a recombinant avian adeno-associated virus (rAAAV) that would specifically express hLYZ in the chicken oviduct and harvested hLYZ from the egg whites of laying hens. The oviduct-specific human lysozyme expression cassette flanked by avian adeno-associated virus (AAAV) inverted terminal repeats (ITRs) was subcloned into the modified baculovirus transfer vector pFBX, and then the recombinant baculovirus rBac-ITRLYZ was generated...
March 2, 2017: Poultry Science
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