adeno associated viral gene delivery in neurodegenerative disease
Báo cáo khoa học: Adeno-associated virus gene transfer in Morquio A disease – effect of promoters and sulfatase-modifying factor 1 pot
... been associated with improvement in polyadenylation ⁄ transport and mRNA processing, which resulted in a sixfold to 50-fold increase in the indicator (CAT) protein [40] The presence of introns in ... promoter silencing does not happen in particular cases, including our study, remains unknown However, in vitro [60] and in vivo [61] studies have shown that AAV vectors induce a change in gene expression ... (2004) Transcriptional silencing is associated with extensive methylation of the CMV promotor following adenoviral gene delivery to muscle J Gene Med 6, 395–404 Prosch S, Stein J, Staak K, Liebenthal...
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Báo cáo khoa học: Nature, nurture and neurology: gene–environment interactions in neurodegenerative disease docx
... Hannan Gene environment interactions in neurodegenerative disease Table Environmental in uences on neurodegenerative disease AD, Alzheimer’s disease; HD, Huntington’s disease; PD, Parkinson’s disease ... variability in brain ageing and associated cognitive 2355 Gene environment interactions in neurodegenerative disease decline in all human populations, forming a template on which specific disease gene ... made in identifying genetic factors contributing to PD in recent years [135] Low concordance for clinical disease in monozygotic twins indicates environmental in uences on PD [136], and the finding...
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Báo cáo sinh học: " The state of the art of adeno-associated virus-based vectors in gene therapy" ppt
... isolation, as well as methods for gene transfer and expression in in vitro studies As this vector system was used with increasing in gene delivery protocols, the interest in the complexities of AAV ... during the last 10 years This increasing interest on AAV is justified by its characteristic features that distinguish it from many other viral vector systems, such as retro/lentiviral and adenoviral ... of expressing foreign genes in mammalian cells [8,9], in the early 1980s, AAV2 vectors have rapidly gained popularity in gene therapy applications, due to some of their distinguishing biological...
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Báo cáo hóa học: " The state of the art of adeno-associated virus-based vectors in gene therapy" doc
... isolation, as well as methods for gene transfer and expression in in vitro studies As this vector system was used with increasing in gene delivery protocols, the interest in the complexities of AAV ... during the last 10 years This increasing interest on AAV is justified by its characteristic features that distinguish it from many other viral vector systems, such as retro/lentiviral and adenoviral ... of expressing foreign genes in mammalian cells [8,9], in the early 1980s, AAV2 vectors have rapidly gained popularity in gene therapy applications, due to some of their distinguishing biological...
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Báo cáo khoa học: Novel brain 14-3-3 interacting proteins involved in neurodegenerative disease doc
... in neurodegenerative disease observed during neurodegeneration in mice as a result of scrapie infection [13] 14-3-3 isoforms play a key role in neurodegeneration in the polyglutamine-repeat disease ... FEBS PDZ binding motif Cadherin Receptor Ca++ Extracellular p120ctn or δ-catenin Intracellular β or γ− catenin α-catenin Actin Filaments 4203 14-3-3 in neurodegenerative disease 14-3-3 Binds endogenous ... the insulin receptor tyrosine kinase substrate protein of 53 kDa (IRSp53) IRSp53 is an SH3 domain-containing adaptor protein originally identified as a substrate of the insulin receptor kinase...
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Báo cáo khoa học: The role of DYRK1A in neurodegenerative diseases docx
... protein at the sites that are phosphorylated in AD In NFTs, tau protein is phosphorylated by several protein kinases, including GSK-3b, cyclin-dependent kinase5 (CDK5), c-Jun N-terminal kinase, ... hyperphosphorylated in the DS brain Gene dosage-related increases in DYRK1A levels in DS appear to be the major factor distinguishing the pattern and consequences of tau protein hyperphosphorylation in DS and in ... total proteins) In all examined subregions of brains of DS subjects, the level of Role of DYRK1A in neurodegenerative diseases DYRK1A is higher than in control brains, but with an increase that...
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gene therapy in cardiovascular disease
... translation into protein Another form of gene blockade is the use FIGURE 8.1 Gene therapy strategies See color insert (A) Gene transfer involves delivery of an entire gene, either by viral infection ... such as vein grafting or transplantation and may represent a means of enhancing plasmid gene delivery Controlling Gene Expression in Cardiovascular Tissue In addition to effective gene delivery, ... resistance in the ischemic rabbit hind limb Applying a similar strategy, 1011 viral particles of an adenoviral vector encoding human FGF-5, containing a secretary signal sequence at its amino terminus,...
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Báo cáo sinh học: " Application of a haematopoetic progenitor cell-targeted adeno-associated viral (AAV) vector established by selection of an AAV random peptide library on a leukaemia cell line" pot
... High-level globin gene expression mediated by a recombinant adenoassociated virus genome that contains the 3' gamma globin gene regulatory element and integrates as tandem copies in erythroid cells ... Gilbert LB, Arap W, Pasqualini R, Weitzman MD: Incorporation of tumor-targeting peptides into recombinant adeno- associated virus capsids Mol Ther 2001, 3:964-975 Nicklin SA, Buening H, Dishart KL, de ... Champion-Arnaud P, David-Ameline J, Moullier P: Factors influencing recombinant adeno- associated virus production Hum Gene Ther 1998, 9:695-706 Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I, Potter M, Chesnut...
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Báo cáo sinh học: "Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice" pptx
... transcriptsin both slow twitch skeletal muscle as well as the heart It binds Ca2+ and prevents actin-myosin interaction in resting muscle Page of (page number not for citation purposes) Genetic Vaccines ... contains cyclic AMP response-element binding protein (CRE-BP – a member of the leucine zipper family of transcription factors) binding sites as well as NFkappaB binding sites The CMV promoter confers ... were assayed using the Galacto-Star chemiluminescence reporter gene assay system (Tropix, Inc., Bedford, MA, USA) Protein concentrations were determined using the Bio-Rad DC protein assay kit (Bio-Rad,...
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Báo cáo sinh học: " In vitro evaluation of a double-stranded selfcomplementary adeno-associated virus type2 vector in bone marrow stromal cells for bone healing" pptx
... However, adenoviral vectors induce strong cellular and humoral immune responses in the host that results in tissue injury and loss of transgene expression [16-18] Recombinant adeno- associated viral ... non-enveloped single-stranded DNA virus [19] This unique viral vector has the distinct advantage of being capable of infecting a wide range of host cell types including dividing and non-dividing cells ... of pre-clinical animal models of bone defect healing [7-10] In previous studies of ex vivo gene therapy in our laboratory, lentiviral and adenoviral mediated over-expression of BMP-2 in rat bone...
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Báo cáo sinh học: " Recombinant adeno-associated virus type 2-mediated gene delivery into the Rpe65-/- knockout mouse eye results in limited rescue" ppsx
... to the reduction in numbers of lipid droplets, the layer of basal infoldings was also thinner in rAAV.RPE65-injected eyes (Fig 6A and 6B) Immunostaining using the anti-SWC opsin antibody demonstrated ... encoding Prph2 in a retinal degeneration slow mouse model [42,43] Vision is maintained through the close and precise interaction between all the retinal cells For example both in humans and in ... characteristics following subretinal injection in the rat Vision Res 2004, 44:339-348 Rabinowitz JE, Rolling F, Li C, Conrath H, Xiao W, Xiao X, Samulski RJ: Cross-packaging of a single adeno- associated...
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Báo cáo y học: "Human, viral or mutant human IL-10 expressed after local adenovirus-mediated gene transfer are equally effective in ameliorating disease pathology in a rabbit knee model of antigen-induced arthritis" ppt
... synovitis in the knee joints was observed In addition, these cytokines displayed not only a chondroprotective effect in blocking cartilage matrix breakdown but also a chondrogenic effect in maintaining ... transfer of vIL-10 gene inhibits murine collagen-induced arthritis J Immunol 1998, 160:5213-5220 34 Yeh P, Perricaudet M: Advance in adenoviral vectors: from genetic engineering to their biology ... encoding interleukin-4 as non -viral vectors for antiinflammatory gene therapy in collagen-induced arthritis J Gene Med 2002, 4:300-307 43 Woods AM, Thompson SJ, Wooley PH, Panayi G, Klavinskis...
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Báo cáo y học: "Complete suppression of viral gene expression is associated with the onset and progression of lymphoid malignancy: observations in Bovine Leukemia Virus-infected sheep" pdf
... mechanisms might be involved in tumor -associated silencing Altogether, our findings strongly support the hypothesis that switching-off viral gene expression, including Tax, the essential contributor ... mutations in other regions might contribute to proviral extinction, our data suggest that tumor -associated silencing in S267 results from molecular mechanisms that are not linked to genetic changes Interestingly, ... documented in HTLV-1 -associated disease and both genetic and epigenetic silencing mechanisms have been described [13,27,28] This study in sheep contributes to the further understanding of tumor-associated...
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Báo cáo y học: "Differential gene expression in HIV/SIV-associated and spontaneous lymphomas"
... the interleukin gene; λIg: the λ-chain of immunoglobulin gene; γIg: the γ-chain of immunoglobulin gene; IFN-ind: the interferon-inducible gene 6-16; IL4R: the interleukin receptor gene; TAP2: ... of immunoglobulin gene, γIg – the γ-chain of immunoglobulin gene, IFN-ind – the interferone-inducible gene 6-16, IL4R – the interleukin receptor gene, TAP2 – the transport protein gene, hnRNP A1 ... oncogene, constant part of the λIg gene, the mitochondrial genes of NADH dehydrogenase subunit (ND4), the interferon-inducible gene 6-16 (Inf-ind), the 16S rRNA gene (most probably the humanin gene...
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Báo cáo y học: " Vgf is a novel biomarker associated with muscle weakness in amyotrophic lateral sclerosis (ALS), with a potential role in disease pathogenesis"
... tissue staining intensities influenced the measured values Average value density measurements from individual Vgf immunoreactive dorsal spinal cord neurons, reflecting immunostaining intensity, ... delimiting the cellular area of interest free hand, using predetermined criteria to define the region of interest The immune intensity of the cellular Vgf encompassing the L3-L5 regions of spinal ... quantified using a CobraII Auto γ Counter (PerkinEl- 94 mer, Wellesley, MA) Adeno- Vgf viral constructs The replication-defective recombinant -adeno- expression virus was generated using the Adeno- X...
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Báo cáo khoa học: Efficient inhibition of b-secretase gene expression in HEK293 cells by tRNAVal-driven and CTE-helicase associated hammerhead ribozymes doc
... pUC-KE-tRNACTE-Rz-2, encoding Rz-1 and Rz-2, respectively, were obtained on a multimilligram scale Constructs containing inactive versions of Rz-1 and Rz-2 were obtained in the same way by using templates ... protein, and in consequence, to reduce the amount of Ab peptide released in neuronal cells We asked the question whether we can inhibit expression of the gene of BACE protein using our engineered ... protease Asp2, also called BACE protein or memapsin [9–13] This protein is involved in proteolytic cleavage of an amyloid precursor protein APP, and directly in uences the release of the toxic...
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Báo cáo khoa học: Structure, expression and regulation of the cannabinoid receptor gene (CB1 ) in Huntington’s disease transgenic mice ppt
... dopamine neurotransmission, and abnormalities in cannabinoid signalling or modulation of dopamine signalling or both have been implicated in a number of neurodegenerative diseases including HD ... protein; RAR, retinoic acid nuclear receptor; WHN, winged helix protein; BKLF, basic krueppel-like factor; ZF5, zinc finger domain; MYT1, zinc finger TF involved in primary neurogenesis; E2A proteins, ... [10] and that neuronal intranuclear inclusions (NIIs) containing the human transgene-encoded amino terminus of human huntingtin form more slowly throughout the brain tissue in R6/1 compared to R6/2...
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Báo cáo khoa học: NovelN,N¢-diacyl-1,3-diaminopropyl-2-carbamoyl bivalent cationic lipids for gene delivery – synthesis,in vitro transfection activity, and physicochemical characterization docx
... lipid)-mediated in vivo gene delivery to mouse liver Gene Ther 10, 180187 20 Narang AS, Thoma L, Miller DD & Mahato RI (2005) Cationic lipids with increased DNA binding afnity for nonviral gene transfer in ... synthesized with the intent of enhancing the transport of exogenous genetic material into cells in vitro, and their design eliminates the need for additional components in the gene- delivery system ... cytofectins for gene delivery M Spelios and M Savva Cationic lipidpDNA binding studies Binding curves of EtBr-intercalated pDNA titrated with aliquots of cationic lipid dispersions are shown in Fig...
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Báo cáo khoa học: Huntington’s disease: revisiting the aggregation hypothesis in polyglutamine neurodegenerative diseases pptx
... through the brain’s inherent plasticity Protein aggregation in neurodegenerative disease is not unique to polyglutamine diseases, and is a common theme with other amyloid diseases, including transmitted ... polyglutamine-containing protein, whether proven to be misfolded or not This is an important distinction, given the role of huntingtin in vesicular interactions [22,23] Even the term ‘huntingtin’ is ... Three groups, including ours, have independently used FRAP in the context of mutant huntingtin exon 1, ataxin-1 and ataxin-3 proteins Some polyglutamine-expanded proteins in puncta can exchange...
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Báo cáo y học: "Gene profiling for defining targets for new therapeutics in autoimmune diseases" potx
... (‘rolling’) Recent in vivo observations have validated the biological relevance of this finding Fucosyltransferase VII was in fact found to be upregulated on T cells infiltrating the inflamed joints ... naïve CD4+ T cells into Th2 cells by inducing the initial production of IL-4 in CD4+ T cells [18] In addition, it has been shown that IL-6 inhibits Th1 differentiation in an IL-4-independent manner ... The finding that genes regulating key steps in the process of Available online http://arthritis-research.com/content/5/2/47 leukocyte extravasation into inflamed tissues are coregulated in human...
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