• Furthermore, the mTORC1 activator (siRNA-TSC2) partially inhibited the above beneficial effects of TSN, suggesting that mTORC1 was the target of TSN to regulate autophagy. (hindawi.com)
  • Notably, inhibition of the CaMKK (calmodulin-dependent protein kinase kinase) had little affect on GLUT translocation, whereas the inhibition or knockdown of AMPK (compound C, siRNA) activity prevented GLUT3 translocation to the cell surface after glutamate excitation. (jneurosci.org)
  • DTx Pharma is developing a means to streamline the delivery of small interfering RNA (siRNA) therapeutics, which target and block gene expression to treat diseases. (ocbj.com)
  • We attach the fatty acids to the siRNA and trick the cell into taking up that RNA so it can get rid of those bad genes that are driving different diseases. (ocbj.com)
  • The trials used the proprietary small interfering RNA (siRNA) method to silence Cbl-b in patient-derived peripheral blood mononuclear cells. (scienceboard.net)
  • Beyond lomitapide, Zirlik pointed out that there are other drugs that have shown potential in managing HoFH and could potentially be used in the pediatric population, such as angiopoietin-like 3 protein (ANGPTL3) inhibitors and small interfering RNA (siRNA) compounds that target upstream production. (medscape.com)
  • Researchers in the laboratory of Mark E. Davis at the California Institute of Technology have been using small interfering RNA (siRNA), sometimes known as silencing RNA, to "silence" specific genes that are implicated in certain malignancies. (news-medical.net)
  • The significance of cadmium chloride-mediated inhibition of eIF4E was further investigated by silencing the expression of eIF4E by employing small interfering RNA (SiRNA) specifically targeting eIF4E. (cdc.gov)
  • They used siRNA or Small Interfering RNA to silence MCJ in the liver. (medicalxpress.com)
  • Theoretically speaking, the nanoparticles would include small, interfering RNA, or siRNA, a class of molecules developed to shut down specific genes. (infoniac.com)
  • RNA interference is an evolutionary conserved gene regulatory mechanism that can be used by introducing exogenous synthetic double-stranded RNAs, so called small interfering RNA (siRNA). (lu.se)
  • siRNAs are sequence-specific inhibitors that are easily designed and could in theory target any gene of interest, making siRNA a promising modality for targeted therapy. (lu.se)
  • However, a key challenge in translating siRNA into the clinic is the inefficacy to deliver siRNA across the plasma membrane, but most importantly, to escape the endosomal system and reach the cytosol where they can interact with the RNA interference machinery. (lu.se)
  • One class of such RNA agents is small interfering RNAs (siRNAs) that mediate viral message degradation with perfect base pairing to the target. (ca.gov)
  • Moreover, combinations with small RNAs capable of turning over multiple target substrates, such as siRNAs and ribozymes, showed greater inhibition to HIV replication in a one-month challenge assay. (ca.gov)
  • We demonstrate the versatility of the MCM7 platform in expressing and efficiently processing the siRNAs as miRNA mimics along with nucleolar small RNAs. (ca.gov)
  • This represents the first efficacious example of combining Drosha-processed siRNAs with small nucleolar ribonucleoprotein (snoRNP)-processed nucleolar RNA chimeras from a single intron platform for effective inhibition of viral replication. (ca.gov)
  • Small interfering RNAs, or siRNAs, could be great targeted treatment tools for breast and other cancers. (vectorblog.org)
  • Though it was first discovered in plants, researchers have known for about a decade that small interfering RNAs (siRNAs) are active in mammals like us, and are already working on ways to harness them for shutting down genes promoting cancer and other diseases. (vectorblog.org)
  • Even better, the team found no evidence of inflammation or toxicity in the liver, kidneys, or bloodstream, which-along with tests showing where the siRNAs accumulated-suggested that the siRNAs were silencing PLK1 only in their intended targets. (vectorblog.org)
  • PLK1 is ubiquitously expressed and is needed for cells to divide," Lieberman notes "but because we targeted the siRNAs only to HER2-expressing tumor cells, we could silence this gene with great specificity and no toxicity to other tissues. (vectorblog.org)
  • They include coding mRNAs and non-coding (nc) RNAs among them antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), micro-RNAs (miRNAs), small activating RNAs (saRNAs), RNA aptamers and RNA guides. (frontiersin.org)
  • There are currently several products containing RNA on the market, and many are under development, among them mRNA, antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), small activating RNAs (saRNAs), micro-RNAs (mi-RNAs), RNA aptamers and RNA guides. (frontiersin.org)
  • Small interfering RNAs (siRNAs) expressed from short hairpin RNAs (shRNAs) are a powerful way to mediate gene specific RNA interference (RNAi) in mammalian cells. (sigmaaldrich.com)
  • Double-stranded RNA in cells is cut by an enzyme called Dicer into small chunks about 21 nucleotides long, referred to as short interfering RNAs (siRNAs). (discovermagazine.com)
  • These siRNAs bind to any RNA molecules with complementary chunks, essentially placing a target on the RNA molecule. (discovermagazine.com)
  • The patent also claims the use of siRNAs that interfere with the ALL response-signaling pathway. (genomeweb.com)
  • Is the Subject Area "RNA interference" applicable to this article? (plos.org)
  • A molecular phenomenon called RNA interference (RNAi) -in which small pieces of RNA silence the expression of individual genes-could provide an alternative solution for breast and other cancers. (vectorblog.org)
  • Asn1 , Ppo5 , PhL , R1 , and VInv ), which results in the reduced level of mRNA transcripts (and subsequently expressed proteins) for those same genes using the RNA interference (RNAi) pathway. (canada.ca)
  • The plant kills its pest by taking advantage of RNA interference (RNAi), which is an innate genetic mechanism to suppress gene expression found in many species. (discovermagazine.com)
  • Besides, biomimetic carriers based on macrophages have been extensively explored to prolong circulation, enhance tumor-targeted delivery, and reduce the immunogenicity of therapeutics to augment therapeutic efficacy. (springer.com)
  • This funding helps to devise new ways to target therapeutics and other molecules to specific brain cell populations. (mordorintelligence.com)
  • SAN DIEGO , Feb. 9, 2017 /PRNewswire/ -- Arcturus Therapeutics, Inc. ("Arcturus" or the "Company"), a leading RNA medicines company, today announced that President & CEO Joseph E. Payne will present a corporate update at the 2017 Annual BIO CEO & Investor Conference on Monday, February 13 th at 1:00 p.m. (prnewswire.com)
  • Arcturus has several partnered and fully funded RNA therapeutics in its pipeline (including HBV, NASH, and orphan diseases), and is appraising investors interested in the Company's wholly owned rare disease messenger RNA programs. (prnewswire.com)
  • Founded in 2013 and based in San Diego , Arcturus Therapeutics, Inc. is an RNA medicines company with enabling technologies - UNA Oligomer chemistry and LUNAR™ nanoparticle delivery. (prnewswire.com)
  • Arcturus' versatile RNA therapeutics platform can be applied toward all types of RNA medicines including small interfering RNA, messenger RNA, antisense RNA, microRNA and gene editing therapeutics. (prnewswire.com)
  • The Company's commitment to the development of novel RNA therapeutics has led to significant partnerships with Janssen Pharmaceuticals , Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical , Inc., and Takeda Pharmaceutical Inc. For more information, visit www.ArcturusRx.com . (prnewswire.com)
  • Zamore, P. D., Tuschl, T., Sharp, P. A. & Bartel, D. P. RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. (nature.com)
  • Embryonic knockdown of the function of homologs of these genes in rat neocortical projection cell progenitors by in utero electroporation of plasmids encoding small hairpin RNA (shRNA) revealed that all three genes disrupted neuronal migration to the neocortex. (nih.gov)
  • Although HMG proteins do not possess intrinsic transcriptional activity, they are called architectural transcription factors because they modulate the transcription of their target genes by altering the chromatin structure at the promoter and/or enhancers 15 . (nature.com)
  • Synthetic antisense RNAs are used to effect the functioning of specific genes for investigative or therapeutic purposes. (genomicglossaries.com)
  • Analysis of the WNK1-dependent transcriptome by RNA-Seq demonstrated that WNK1 regulates the expression of 1858 genes during decidualization. (bioone.org)
  • With the exception of late blight resistance, the other novel traits (i.e. reduced asparagine levels, lower levels of reducing sugars, and reduced black spot bruising) are achieved through the transcription of inverted repeat sequences containing small fragments of DNA from five different endogenous genes (i.e. (canada.ca)
  • The German team created potato plants that produce dsRNAs designed to target two genes in the beetle that are absolutely essential for survival (β-actin and Shrub). (discovermagazine.com)
  • Arcturus' proprietary UNA technology can be used to target any gene in the human genome, as well as viral genes, and other species for therapeutic purposes. (prnewswire.com)
  • To identify the specific binding of target genes, RIP and pull-down assays were employed. (bvsalud.org)
  • A β-hairpin in the L1 domain secures the t9-t13 segment and coordinates t9-t13 docking with extended guide-target pairing. (nature.com)
  • The invention relates to "small interfering RNAs or small hairpin RNA and compositions comprising same … that target human cyclophilin A to inhibit hepatitis C infection," according to the patent's abstract. (genomeweb.com)
  • More specifically, antisense oligonucleotides that are useful as reagents for target validation , or as drugs, are engineered molecules that differ from natural RNA but that have a base sequence that is recognized as being complementary to a very specific mRNA sequence. (genomicglossaries.com)
  • Because alternative RNA splicing allows different types of mRNA molecules to be created from a single gene, it generates the diversity of protein function and structure that is essential to complex organisms. (genomicglossaries.com)
  • RNA molecules which hybridize to complementary sequences in either RNA or DNA altering the function of the latter. (genomicglossaries.com)
  • Cells have been trained for billions of years to reject foreign RNA molecules because viruses inject RNA into the body," CEO Artie Suckow told the Business Journal. (ocbj.com)
  • A set of cellular components referred to as RNA-induced silencing complex then zero in on these RNA molecules and destroy them. (discovermagazine.com)
  • Due to the hydrophilic (non-fat-soluble) nature of the molecules, they have been formulated as lipid nanoparticles to aid transport into cells or conjugated to molecules with an ability to target certain cells in the body (eg, hepatocytes) to aid specificity of action. (bmj.com)
  • Currently, most leading therapeutic drugs undergoing clinical trials for NASH are small molecules given as a systemic treatment," Rincon said. (medicalxpress.com)
  • DARPA considers that tiny, autonomous drug delivery systems would be much more effective , being able to send medicated molecules straight into a targeted cell. (infoniac.com)
  • Primary human endometrial stromal cells (HESCs) were subjected to small interfering RNA knockdown of WNK1 followed by in vitro decidualization. (bioone.org)
  • Furthermore, acute knockdown by intrathecal injection of Tmem45b small interfering RNA, either before or after inflammation induction, successfully reduced mechanical pain hypersensitivity. (iasp-pain.org)
  • RNA-like oligonucleotides that are complementary to a portion of a target mRNA molecule. (genomicglossaries.com)
  • The Medicines Manufacturing Innovation Centre launched a collaboration with AstraZeneca, Exactmer, Novartis, and UK Research and Innovation to develop a scalable, sustainable, and more cost-effective medicines manufacturing process for oligonucleotides: short strands of synthetic DNA or RNA. (mordorintelligence.com)
  • Endogenous MCM7 MicroRNA Cluster as a Novel Platform to Multiplex Small Interfering and Nucleolar RNAs for Combinational HIV-1 Gene Therapy. (ca.gov)
  • In this current study, we utilized an endogenous microRNA (miRNA) cluster as a platform to express multiple anti-HIV small RNAs. (ca.gov)
  • Endogenous antisense RNAs function as regulators of gene expression by a variety of mechanisms. (genomicglossaries.com)
  • Here we explore combinations of different classes of therapeutic anti-HIV-1 RNAs expressed from within the context of an intronic MCM7 (minichromosome maintenance complex component-7) platform that naturally harbors 3 microRNAs (miRNAs). (ca.gov)
  • The compositions comprise oligomeric compounds targeted to small non-coding RNAs and pri-miRNAs. (genomeweb.com)
  • Finally, anti-HIV RNA decoys that aim to titrate out early essential viral replication proteins Tat and Rev are constructed. (ca.gov)
  • Argonaute (AGO) proteins use small RNAs to recognize transcripts targeted for silencing in plants and animals. (nature.com)
  • Anti-RNP antibodies are directed against the U1 particle of an extractable nuclear antigen (ENA) named ribonucleoprotein,[9] which is made up of a complex of proteins and a small nuclear RNA named U1. (medscape.com)
  • [ 9 ] which is made up of a complex of proteins and a small nuclear RNA named U1. (medscape.com)
  • A complementary RNA sequence that binds to a naturally occurring (sense) mRNA molecule, thus blocking its translation. (genomicglossaries.com)
  • The mammalian target of rapamycin (mTOR) is an evolutionarily highly conserved intracellular serine/threonine kinase family member. (hindawi.com)
  • Abstract Combinational therapy with small RNA inhibitory agents against multiple viral targets allows efficient inhibition of viral production by controlling gene expression at critical time points. (ca.gov)
  • Highly specific and effective gene silencing of any disease can be achieved by an accurate knowledge of the target mRNA sequence and rational design of its complementary antisense agents for the downregulation of its protein message. (genomicglossaries.com)
  • The compositions of the … invention are highly potent, thereby allowing effective knock-down of a specific target protein at relatively low doses. (genomeweb.com)
  • The researchers focused on MCJ or Methylation-Controlled J Protein as a target for NASH. (medicalxpress.com)
  • RNA methyltransferase nucleolar protein p120 (NOP2), commonly referred to as NOP2/Sun RNA methyltransferase family member 1 (NSUN1), is involved in cell proliferation and is highly expressed in various cancers. (bvsalud.org)
  • It also is a direct target gene of the Wnt pathway and is upregulated by beta-catenin. (wikipedia.org)
  • In summary, TSN reduces podocyte damage induced by high glucose via inhibiting mTORC1 pathway and downstream targets and restoring podocyte autophagy. (hindawi.com)
  • After second- strand cDNA synthesis, T7 RNA polymerase was used to generate aRNA. (genomicglossaries.com)
  • Treatment wth olpasiran, a small interfering RNA product targeting lipoprotein (a) synthesis, led to a profound and sustained reduction in Lp(a) concentration when administered every 12 weeks in a new phase 2 study. (medscape.com)
  • Lead interferes with the synthesis of heme, resulting in accumulation of ALA in tissues and elevated excretion of ALA in urine, elevation of zinc protoporphyrin in erythrocyte, reductions in blood hemoglobin, and in a hypochromic, normocytic anemia at higher levels of exposure. (cdc.gov)
  • 87(5): 1663-1667] Specifically, the investigators started by priming whole cerebellar RNA with a synthetic oligonucleotide containing a T7 RNA polymerase promoter sequence. (genomicglossaries.com)
  • Founded earlier this year, Apeiron Biologics' spinout invIOs has a proprietary cell therapy platform for the modulation of intracellular immuno-oncology targets that enables rapid treatment of cancer patients using their own immune cells with short out-of-body time in out-patient settings. (scienceboard.net)
  • Gene therapy approaches are attractive alternatives where the most vulnerable immune cells are modified with antiviral small RNAs with the goal to engineer an HIV-resistant immune system. (ca.gov)
  • Moreover, we demonstrated enrichment/selection for cells expressing levels of the antiviral RNAs that provide optimal inhibition under the selective pressure of HIV. (ca.gov)
  • Virus entry and replication strategies are potential targets for antiviral drug treatments. (cdc.gov)
  • Structure-based cleavage mechanism of Thermus thermophilus Argonaute DNA guide strand-mediated DNA target cleavage. (nature.com)
  • Nucleation, propagation and cleavage of target RNAs in Ago silencing complexes. (nature.com)
  • As they need to be composed of an exact complementary sequence to be able to cleave the target mRNA, they are thought to have few unwanted effects apart from infusion or injection site reactions. (bmj.com)
  • The other class of anti-HIV RNAs is self-catalytic ribozymes that recognizes their target by base pairing following by enzymatic cutting of the target. (ca.gov)
  • Fig. 3: Catalytic-competent conformation of the AtAgo10-guide-target complex. (nature.com)
  • RNA which contains an intron sequence that has an enzyme- like catalytic activity. (genomicglossaries.com)
  • The lentiviral transduction particles are produced from an shRNA lentiviral non-target control plasmid. (sigmaaldrich.com)
  • It is useful as a negative control in experiments with the MISSION shRNA target sets. (sigmaaldrich.com)
  • 10] Ribonucleoprotein is part of the splicosome and also includes Smith (Sm) antigen, which is the target of the anti-Smith antibody and is frequently positive in SLE. (medscape.com)
  • While the first mRNA vaccines against an infectious disease have reached the market ( 1 , 2 ), many other medicinal products with RNA as an active substance (see Table 1 ), either vaccines against non-infectious diseases or non-vaccine drugs, are under development ( 3 - 18 ) or even approved ( 19 - 25 ). (frontiersin.org)
  • If not for oxalate's high affinity for calcium and the low solubility of calcium oxalate, oxalate and oxalate metabolism would be of little interest. (medscape.com)
  • In particular, targeted therapy that can address genetic changes would be of great value. (lu.se)
  • Here we present cryogenic electron microscopy structures of the Arabidopsis thaliana Argonaute10 (AtAgo10)-guide RNA complex with and without a target RNA representing a slicing substrate. (nature.com)
  • In particular, advanced high resolution microscopy techniques have been used to in detail characterize and determine the efficacy of lipid mediated delivery of RNA. (lu.se)
  • Viral proteases (HCV and SARS-CoV) and viral RNA-dependent RNA polymerases (Lassa virus, HCV, WNV) are major targets, but treatments targeting viral enzymes often induce the rapid emergence of drug-resistant viruses. (cdc.gov)
  • In its binding with sulfhydryl groups, lead may interfere with the activity of zinc metalloenzymes, as zinc binds to a sulfhydryl group at the active site. (cdc.gov)
  • Although hemolysis and lipemia do not interfere with this assay, they are best avoided. (medscape.com)
  • While targeted drugs like trastuzumab (Herceptin) and lapatinib (Tykerb) have made these tumors easier to treat, those that resist these drugs, relapse or don't have HER2 on their cells' surfaces can still stymie oncologists. (vectorblog.org)
  • Also, developing therapeutic COVID-19 medicines employing essential mutational targets, such as the 5'-polyU tract, can lessen the production and spread of new SARS-CoV-2. (mordorintelligence.com)
  • Therefore, with the aim to summarize the current knowledge on the initiation of the atherosclerotic process, in this paper, we review the early markers of atherosclerosis and we address the main therapeutic targets for preventing atheroma formation at its very initial stages focusing on inflammation, oxidative stress, endothelial dysfunction, and the interaction between platelets and endothelium. (hindawi.com)
  • Komohara Y, Fujiwara Y, Ohnishi K, Takeya M. Tumor-associated macrophages: potential therapeutic targets for anti-cancer therapy. (springer.com)
  • Our results thus suggest that eIF4E is a cellular target for cadmium toxicity and that the cadmium -induced cytotoxicity and cell death may be due to the inhibition of eIF4E. (cdc.gov)
  • Researchers at Stanford University used such a method to produce amplified heterogeneous populations of RNA from limited quantities of cDNA. (genomicglossaries.com)
  • More recently, another group of researchers reported that they had developed a process for optimizing low- abundance RNA, by combining aRNA amplification with template- switching. (genomicglossaries.com)
  • In an effort to combat a growing worldwide epidemic of Nonalcoholic Fatty Liver Disease (NAFLD), scientists have discovered a new target and a new therapy that has shown promising results in preclinical mouse models, according to researchers at the University of Colorado Anschutz Medical Campus. (medicalxpress.com)
  • We demonstrated persistent expression of each anti-HIV RNAs in CEM T cell lines that stably integrate the transgene. (ca.gov)
  • This is mediated by cytokines and other mediators released by the activated immune cells invading the islets, which activate secondary pathways of cell death in the target beta cells. (europa.eu)
  • This Specific Targeted Research Project (STREP) aims to utilise functional genomics to identify pathways responsible for the reduction of beta cell mass in diabetes, and use this knowledge to define targets for intervention to preserve beta cell mass. (europa.eu)
  • This step should identify and validate targets to preserve beta cell mass in diabetes. (europa.eu)
  • The aim of this thesis was to develop novel methods to study the process of endosomal escape and cytosolic delivery of RNA. (lu.se)
  • This thesis advances our understanding on the limiting step of endosomal escape and cytosolic entry of RNA during lipid-based delivery. (lu.se)
  • However, things are becoming more and more complicated as the current evolution of technology in this field makes it possible to produce similar RNA-based drugs using different approaches. (frontiersin.org)
  • Plant small RNAs: advancement in the understanding of biogenesis and role in plant development. (nature.com)
  • Results show that prokaryotic and eukaryotic AGOs use distinct mechanisms for achieving target slicing and provide insights into small interfering RNA potency. (nature.com)
  • These data suggest survivin might provide a new target for cancer therapy that would discriminate between transformed and normal cells. (wikipedia.org)
  • Interestingly, cells with optimal level of small RNA expression to effectively inhibit viral replication were enriched under the selective pressure of HIV, demonstrating the importance of balancing expression and functionality for successful gene therapy applications. (ca.gov)
  • The combinations of si/snoRNAs represent a new paradigm for combinatorial RNA-based gene therapy applications. (ca.gov)
  • Yang QY, Guo NN, Zhou Y, Chen JJ, Wei QC, Han M. The role of tumor-associated macrophages (TAMs) in tumor progression and relevant advance in targeted therapy. (springer.com)
  • This may guide targeted therapy in CRC patients. (bvsalud.org)
  • According to the European Union (EU) legislation, these products can be currently categorized into different regulatory statuses, depending, for vaccines, on their target (infectious disease or not) and, for other drugs, on how they are obtained (chemically or biologically). (frontiersin.org)
  • Few regulatory studies are available to assist in the regulatory categorization of RNA-based medicinal products. (frontiersin.org)
  • Inflammation and immunity are key factors for the development and complications of atherosclerosis, and therefore, the whole atherosclerotic process is a target for diagnosis and treatment. (hindawi.com)
  • Many RNA-based drugs, both vaccines and non-vaccines, are under development or even approved. (frontiersin.org)
  • Examples of RNA-based drugs currently or formerly on the market or under development. (frontiersin.org)
  • Fig. 2: AtAgo10-guide-target complex captured in two functional states. (nature.com)
  • 2017. Long noncoding RNAs and sulforaphane: a target for chemoprevention and suppression of prostate cancer. . (oregonstate.edu)
  • Thus, our study demonstrates that Tmem45b is essential for inflammation- and tissue injury-induced mechanical pain hypersensitivity and highlights Tmem45b as a therapeutic target for future treatment. (iasp-pain.org)
  • The treatment, called DTx-1252, targets the gene responsible for that class of CMT, called CMT1A. (ocbj.com)
  • This multiplex approach reduces the likelihood of viral resistance and enhances the synergistic potential between inhibitory agents with different mechanism of action and targets, with the overall goal of achieving long-term protection against HIV. (ca.gov)
  • Methods for accurately and precisely sampling and quantifying viral RNA in wastewater that are representative of viral shedding within a community are available and continue to be improved. (cdc.gov)
  • Eukaryotic translation initiation factor 4E (eIF4E) is a cellular target for cadmium toxicity. (cdc.gov)
  • Presently, we have investigated whether the translational proto-oncogene, eukaryotic initiation factor 4E (eIF4E) is a cellular target for cadmium toxicity and carcinogenesis. (cdc.gov)