... during which she was involved in some of the first pre-clinical and clinical studies of cationic lipid mediated gene therapy ... Natasha Jane Caplen is a British-American geneticist who discovered RNA interference (RNAi) in mammalian cells. She is a senior ... where she initially conducted studies investigating hybrid viral vector systems. It was while at NHGRI that Caplen developed a ... Caplens postdoctoral training began at St Marys Hospital Medical School where she focused on the development of gene therapy ...

A promising strategy to specifically target viral infections is to suppress viral replication through RNA interference (RNAi). ... which is present in the genomic as well as in all subgenomic RNAs. In assays with infectious SARS-CoV-2, it reduced replication ... Hence, we designed eight small interfering RNAs (siRNAs) targeting the highly conserved 5′-untranslated region (5′-UTR) of SARS ... Berkhout, B. Toward a Durable Anti-HIV Gene Therapy Based on RNA Interference. Ann. N. Y. Acad. Sci. 2009, 1175, 3-14. [Google ...

Endocytosis in gene therapy with non-viral vectors.. Wiener Medizinische Wochenschrift 166, 227-235.. Perng, Y.C., Lenschow, D. ... transgene expression by RNA interference-mediated silencing of interferon. receptors. J. Gene Med. 12, 739-746.. Unterholzner, ... and innate host responses to viral gene therapy vectors. Mol. Ther. 18, 1422-1429.. Sørensen, L.N., Paludan, S.R., 2004. ... gene therapy.. Many previous attempts to improve gene therapy via inhibition of. the IIR have focused on TLR9 (Unterholzner, ...

Applications of lentiviral vectors for biology and gene therapy of neurological disorders.. Lundberg, C., Björklund, T., ... Dose Optimization for Long-term rAAV-mediated RNA Interference in the Nigrostriatal Projection Neurons.. Ulusoy, A., Sahin, G. ... Expression of Multiple Functional RNAs or Proteins from One Viral Vector.. Björklund, T., 2016, In: Methods in Molecular ... Optimized adeno-associated viral vector-mediated striatal DOPA delivery restores sensorimotor function and prevents dyskinesias ...

These results indicate that viral delivery of small interfering RNAs can be used for tumor-specific gene therapy to reverse the ... Recently, we developed a vector that mediates suppression of gene expression through RNA interference. Here, we use a ... Stable suppression of tumorigenicity by virus-mediated RNA interference. DSpace/Manakin Repository. ... retroviral version ... read more of this vector to specifically and stably inhibit expression of only the oncogenic K-RASV12 ...

Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including ... Evaluation of recombinant alphaviruses as vectors in gene therapy. abstract：:Alphavirus vectors based on Sindbis virus and ... Therapeutic RNA interference (RNAi) has emerged as a promising approach for the treatment of many incurable diseases, including ... Particle-mediated gene transfer into murine livers using a newly developed gene gun. abstract：:Although particle-mediated gene ...

RNA Interference in Animal Models. In M Sohail, editor. Gene Silencing by RNA Interference: Technology and Application, London ... Harnessing RNA Interference for Therapy. J. Lieberman & Dykxhoorn, D. 2005. The Silent Revolution: RNA interference as a basic ... Sustained Small Interfering RNA-Mediated Human Immunodeficiency Virus Type 1 Inhibition in Primary Macrophages. J Virol 77:7174 ... She is also working to develop an oral HIV vaccine using Listeria monocytogenes as a vector. Her interest in HIV led to recent ...

Development of viral vectors or related reagents to probe or manipulate cell, transcript, or enhancer functions. ... Some single cell technologies in use include single cell RNA-sequencing (scRNA-seq), single nucleus RNA-sequencing (snRNA-seq ... Antiretroviral therapy (ART) has, in large part, transformed the HIV epidemic into a chronic manageable disease in the United ... However, little is known about 1. the effects of persistent HIV infection or HIV treatment regimens on gene expression in ...

Attenuation and regulation by small RNA molecules, Gene silencing and RNA interference, antisense RNA) Plasmids as cloning ... Genetic transformation of plants; Agrobacterium mediated transfer; Ti plasmid technology; Direct gene transfer methods; Vectors ... Physiological Aspects and Novel Therapies. Cancer Classification Based on Origin, Markers and Histology; Mechanisms of Viral ... Regulatory RNA Biology ( Riboswitch, Regulate gene expression by noncoding RNA, ...

... mediated. Noncoding RNA (ncRNA) is an important regulator involved in gene expression and can be detected in the cytoplasm or ... Some respiratory viral infections can aggravate allergic rhinitis, such as a respiratory syncytial virus (RSV) and human ... In this review, we summarized the interactions of noncoding RNAs and viruses in the occurrence and development of AR, along ... metapneumovirus (hMPV). However, the interaction between viral infection and allergy is complex and the mechanism is still ...

Background/aims: RNA interference (RNAi), a sequence-specific gene silencing technology triggered by small interfering RNA ( ... of viral entry receptor CD81 using a human hepatic cell line and primary B lymphocytes transduced with siRNA-expressing vectors ... Conclusion: Both human and mouse hepatic cells exchange small silencing RNAs, partially mediated by shuttling of exosomes. ... Transmission of siRNA potentially extends the therapeutic reach of RNAi-based therapies against HCV as well as other liver ...

... and kits comprising small interfering RNA that are useful for inhibition of viral-mediated gene expression," the patent ... Title: RNA Interference-Mediated Inhibition of Respiratory Syncytial Virus Expression Using Short Interfering Nucleic Acid ... DNA polynucleotides, plasmids, and viral or non-viral vectors are also provided that encode siRNA or shRNA molecules, which may ... The ... invention also provides methods for selecting a preferred therapy for a particular subject or group of subjects or ...

Microwell Array-Mediated Delivery of Lipoplexes Containing Nucleic Acids for Enhanced Therapeutic Efficacy. In: RNA ... including heavy reliance on viral vectors, and the highly stochastic nature of current transfection methodologies. New ... Gene Delivery to Cultured Embryonic Stem Cells Using Nanofiber-Based Sandwich Electroporation. Anal Chem 2013; 85:1401-1407. ... On-chip clonal analysis of glioma stem cell motility and therapy resistance. Nano Letters 2016. DOI: 10.1021/acs.nanolett. ...

Current cancer therapy strategies are based in surgery, radiotherapy and chemotherapy, being the chemotherapy the one that ... the developed FA-modified Au DENPs may be used as a promising non-viral vector for safe and targeted gene therapy applications. ... Covalent AuNP : The application of RNA interference (RNAi) using AuNPs mainly involves delivery of microRNAs. (miRNAs) and ... GOLD NANOPARTICLE-MEDIATED DRUG DELIVERY. Cytotoxicity of gold nanoparticles is inacceptable level as gold nanoparticles are ...

Our group works on RNA interference, immune signalling pathways and virus/host interactions in arthropod vectors by using ... In particular, we focus on both the viral and host determinants that affect response to therapy and viral pathogenesis. ... We also work on Wolbachia-mediated inhibition of RNA virus replication in insect cells. ... horizontal gene transfer, dUTPase, signalling, viral replication, dUTP. ...

Therefore, the development of a safe and effective disease-modifying therapy that limits or prevents the accumulation of the ... vector delivery and cellular transduction). Furthermore, many of the above-discussed strategies involving RNA interference ... In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration. Mol. Ther. J. Am. Soc. Gene Ther. 2010, 18 ... Viral-vector delivery rodent model (e.g., AAV-hA53T). *. High construct and face validity: area-specific alpha-synuclein ...

Elbashir, S., Lendeckel, W., and Tuschl, T. (2001) RNA interference is mediated by 21-and 22-nucleotide RNAs. Genes Dev 15, 188 ... Nayak, S. and Herzog, R. W. (2009) Progress and prospects: immune responses to viral vectors. Gene Ther 17, 295-304. ... Anson, D. S.(2004) The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and ... Glover, D. J., Lipps, H. J., and Jans, D. A. (2005) Towards safe, non-viral therapeutic gene expression in humans. Nat Rev ...

These enhanced AAV vectors have great potential in gene therapy applications.. Assuntos. Proteínas do Capsídeo/genética , ... RNA interference (RNAi) is a more promising approach for antiviral therapy. Here, 17 single artificial microRNAs (amiRNAs) ... Single point mutation in adeno-associated viral vectors -DJ capsid leads to improvement for gene delivery in vivo. ... which mediates efficient gene expression both in vitro and in vivo. AAV2 and AAV8 are the closest parental vectors of AAV-DJ ...

... are one of the leading vectors for gene therapy applications that deliver gene-editing enzymes, antibodies, and RNA ... interference molecules to eliminate viral reservoirs that fuel persistent infections. As long-lived viral DNA within specific ... Thus, over the past few decades, extensive efforts to optimize AAV vectors for gene therapy applications have focused on capsid ... the most recent strategies to identify AAV vectors with enhanced tropism and transduction in cell types that harbor viral ...

... by transfection of short hairpin RNA expression plasmids against sCLU (sCLU-shRNA) (in vivo) or small interference RNA (sCLU- ... sCLU gene silence can inhibit invasion and growth of LSCC. sCLU may provide a potential therapeutic target against human LSCC. ... Terminal deoxytransferase-mediated dUTP nick end labeling (TUNEL) staining was used to observe the apoptosis in the xenografts ... Furthermore, siRNA-mediated down-regulation of sCLU expression decreases in vitro cell migration and invasion ability. In vivo ...

Prime editors are efficiently delivered to mouse organs with AAV vectors. ... prime editing guide RNA stability and modulation of DNA repair. The resulting dual-AAV systems, v1em and v3em PE-AAV, enable ... Here we describe the identification of bottlenecks limiting adeno-associated virus (AAV)-mediated prime editing in vivo and the ... development of AAV-PE vectors with increased PE expression, ... of an intein-mediated split-Cas9 system for gene therapy. ...

Administration of gene therapy viruses into skeletal muscle, where distal terminals of motor and sensory neurons reside, has ... Administration of gene therapy viruses into skeletal muscle, where distal terminals of motor and sensory neurons reside, has ... This route is minimally invasive and therefore clinically relevant for gene therapy targeting to peripheral nerve soma. For ... In this review article, we outline key characteristics of major gene therapy viruses-adenovirus, adeno-associated virus (AAV), ...

Lambeth, L. S., and Smith, C. A. (2013). Short hairpin RNA-mediated gene silencing. Methods in Molecular Biology, 942, 205-32. ... Ellis, J. (2005). Silencing and variegation of gammaretrovirus and lentivirus vectors. Human gene therapy, 16, 1241-6. doi: ... 2016). CRISPR Interference Efficiently Induces Specific and Reversible Gene Silencing in Human iPSCs. Cell Stem Cell, 18, 541- ... 2006). A lentiviral RNAi library for human and mouse genes applied to an arrayed viral high-content screen. Cell, 124, 1283-98 ...

In conclusion, RNA interference technology was used to establish a stably SPRY2-silenced MM cell line. The results suggested ... To the best of our knowledge, the effects of miR-21-mediated downregulation of SPRY2 gene expression on the proliferation and ... Otsu, Japan). Lentiviral vector LV-anti-miR-21 and control vector were from Shanghai SBO Medical Biotechnology Co. (Shanghai, ... The viral supernatant was replaced with appropriate medium, and 24-48 h after infection, green fluorescence was observed under ...

Modulation of human dendritic-cell function following transduction with viral vectors: implications for gene therapy. Blood 105 ... Cao T, Heng BC (2005). Intracellular antibodies (intrabodies) versus RNA interference for therapeutic applications. Ann Clin ... Illés A, Enyedi B, Tamás P, Balázs A, Bogel G, Melinda, Lukács, Buday L (2006). Cortactin is required for integrin-mediated ... Transcriptional targeting of dendritic cells for gene therapy using the promoter of the cytoskeletal protein fascin. Gene Ther ...

However the clinical application of siRNA-based gene therapy is limited due to the absence of an optimised gene delivery vector ... Therefore, preventing the expression of muHTT by harnessing the specificity of the RNA interference (RNAi) pathway is a key ... Modified cyclodextrins as novel non-viral vectors for neuronal siRNA delivery: focus on Huntingtons disease. (University ... While the model was suitable for assessing nanoparticle-mediated gene knockdown, prostate cancer cells demonstrated a phenotype ...

This unique ability to turn unresponsive cold tumors into responsive hot tumors gives them great potential in GC therapy. ... It also exhaustively introduces the concrete mechanism of invading GC cells and the viral genome composition of adenovirus and ... This unique ability to turn unresponsive cold tumors into responsive hot tumors gives them great potential in GC therapy. ... It also exhaustively introduces the concrete mechanism of invading GC cells and the viral genome composition of adenovirus and ...

The invention is directed at compositions and techniques for selectively reducing expression of a target genes gene product in ... a cell as well as treating diseases that are caused by gene expression ... including the use of RNA interference (RNAi), antisense oligonucleotides (ASOs), and gene editing technologies such as CRISPR/ ... or use plasmids or viral expression vectors for longer-term silencing.. Nucleases are responsible for the degradation of siRNAs ...

... the RNA interference silencing complex, or RISC) to destroy the viral RNA. By introducing siRNAs complementary to specific ... The use of non-integrating plasmid vectors removes the risk of mutagenesis caused by some viral vectors. ... The use of siRNA as an antiviral agent involves a relatively straightforward attack on one or more key viral genes and should ... Most of these candidate vaccines consist of either or both of the RSV surface glycoproteins that mediate membrane fusion (F) ...