Arcturus Therapeutics
Arcturus Therapeutics Holdings Inc. is an American RNA medicines biotechnology company focused on the discovery, development and commercialization of therapeutics for rare diseases and infectious diseases. Arcturus has developed proprietary lipid nanoparticle RNA therapeutics for nucleic acid medicines including small interfering RNA (siRNA), messenger RNA (mRNA), gene editing RNA, DNA, antisense oligonucleotides, and microRNA. The company develops RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. Vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the preclinical development phase. The vaccine for COVID-19 was authorised in Japan in 2023 and the European Union in 2025. History Founded in 2013 by Joseph Payne and Pad Chivukula, Arcturus Therapeutics is headquartered in San Diego, California, USA. Initially, they developed a proprietary lipid nanop ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Public Company
A public company is a company whose ownership is organized via shares of share capital, stock which are intended to be freely traded on a stock exchange or in over-the-counter (finance), over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (listing (finance), listed company), which facilitates the trade of shares, or not (unlisted public company). In some jurisdictions, public companies over a certain size must be listed on an exchange. In most cases, public companies are ''private'' enterprises in the ''private'' sector, and "public" emphasizes their reporting and trading on the public markets. Public companies are formed within the legal systems of particular states and so have associations and formal designations, which are distinct and separate in the polity in which they reside. In the United States, for example, a public company is usually a type of corporation, though a corporation need not be a public company. In the United Kin ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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MicroRNA
Micro ribonucleic acid (microRNA, miRNA, μRNA) are small, single-stranded, non-coding RNA molecules containing 21–23 nucleotides. Found in plants, animals, and even some viruses, miRNAs are involved in RNA silencing and post-transcriptional regulation of gene expression. miRNAs base-pair to complementary sequences in messenger RNA (mRNA) molecules, then silence said mRNA molecules by one or more of the following processes: * Cleaving the mRNA strand into two pieces. * Destabilizing the mRNA by shortening its poly(A) tail. * Reducing translation of the mRNA into proteins. In cells of humans and other animals, miRNAs primarily act by destabilizing the mRNA. miRNAs resemble the small interfering RNAs (siRNAs) of the RNA interference (RNAi) pathway, except miRNAs derive from regions of RNA transcripts that fold back on themselves to form short stem-loops (hairpins), whereas siRNAs derive from longer regions of double-stranded RNA. The human genome may encode ov ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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University Of Texas MD Anderson Cancer Center
The University of Texas MD Anderson Cancer Center (colloquially MD Anderson Cancer Center) is a comprehensive cancer center and autonomous university of the University of Texas System in Houston, Texas. It is the largest cancer center in the world and one of the original three NCI-designated comprehensive cancer centers in the country. It is both a degree-granting academic institution and a cancer treatment and research center located within the Texas Medical Center, the largest medical center and life sciences destination in the world. MD Anderson Cancer Center has consistently ranked #1 among the best hospitals for cancer care and research in the U.S. and worldwide, and it has held the #1 position 20 times in the last 23 years in ''U.S. News & World Report's'' Best Hospitals rankings for cancer care. As of 2023, MD Anderson Cancer Center is home to the highest number of cancer clinical trials in the world and has received more NCI-funded projects than any other U.S. instit ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Salk Institute For Biological Studies
The Salk Institute for Biological Studies is a scientific research institute in the La Jolla community of San Diego, California. The independent, non-profit institute was founded in 1960 by Jonas Salk, the developer of the polio vaccine; among the founding consultants were Jacob Bronowski and Francis Crick. Construction of the research facilities began in spring of 1962. The Salk Institute consistently ranks among the top institutions in the US in terms of research output and quality in the life sciences. As of October 2020, the Salk Institute employs 850 researchers in 60 research groups and focuses its research in three areas: molecular biology and genetics; neurosciences; and plant biology. Research topics include aging, cancer, diabetes, birth defects, Alzheimer's disease, Parkinson's disease, AIDS, and the neurobiology of American Sign Language. March of Dimes provided the initial funding and continues to support the institute. Research is funded by a variety of public sour ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Urea Cycle Disorder
The urea cycle (also known as the ornithine cycle) is a cycle of biochemical reactions that produces urea (NH2)2CO from ammonia (NH3). Animals that use this cycle, mainly amphibians and mammals, are called ureotelic. The urea cycle converts highly toxic ammonia to urea for excretion. This cycle was the first metabolic cycle to be discovered by Hans Krebs and Kurt Henseleit in 1932, five years before the discovery of the TCA cycle. The urea cycle was described in more detail later on by Ratner and Cohen. The urea cycle takes place primarily in the liver and, to a lesser extent, in the kidneys. Function Amino acid catabolism results in waste ammonia. All animals need a way to excrete this product. Most aquatic organisms, or ammonotelic organisms, excrete ammonia without converting it. Organisms that cannot easily and safely remove nitrogen as ammonia convert it to a less toxic substance, such as urea, via the urea cycle, which occurs mainly in the liver. Urea produced by the liv ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Citrin
Citrin, also known as solute carrier family 25, member 13 (citrin) or SLC25A13, is a protein which in humans is encoded by the ''SLC25A13'' gene. Citrin is associated with type II citrullinemia and neonatal intrahepatic cholestasis caused by citrin deficiency (NICCD). Function Citrin (74 kDa) is a dimeric calcium-activated glutamate/aspartate carrier found in the mitochondrial membrane of mammals. Citrin is one of two isoforms of these mitochondrial calcium-activated glutamate/aspartate carriers found in humans and is predominately expressed in non-excitable tissues. Upon binding calcium, citrin catalyzes the transport of glutamate and a proton into the mitochondrial matrix in exchange for aspartate transport to the cytosol. Upon being transported by citrin from the mitochondrial matrix to the cytosol, aspartate is converted into oxaloacetate, and then into malate, which is then transported back into the matrix by means of the malate-aspartate shuttle. Upon entering the mito ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Messenger RNA
In molecular biology, messenger ribonucleic acid (mRNA) is a single-stranded molecule of RNA that corresponds to the genetic sequence of a gene, and is read by a ribosome in the process of synthesizing a protein. mRNA is created during the process of transcription, where an enzyme (RNA polymerase) converts the gene into primary transcript mRNA (also known as pre-mRNA). This pre-mRNA usually still contains introns, regions that will not go on to code for the final amino acid sequence. These are removed in the process of RNA splicing, leaving only exons, regions that will encode the protein. This exon sequence constitutes mature mRNA. Mature mRNA is then read by the ribosome, and the ribosome creates the protein utilizing amino acids carried by transfer RNA (tRNA). This process is known as translation. All of these processes form part of the central dogma of molecular biology, which describes the flow of genetic information in a biological system. As in DNA, genetic inf ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Ornithine Transcarbamylase
Ornithine transcarbamylase (OTC) (also called ornithine carbamoyltransferase) is an enzyme () that catalyzes the reaction between carbamoyl phosphate (CP) and ornithine (Orn) to form citrulline (Cit) and phosphate (Pi). There are two classes of OTC: anabolic and catabolic. This article focuses on anabolic OTC. Anabolic OTC facilitates the sixth step in the biosynthesis of the amino acid arginine in prokaryotes. In contrast, mammalian OTC plays an essential role in the urea cycle, the purpose of which is to capture toxic ammonia and transform it into urea, a less toxic nitrogen source, for excretion. Reaction mechanism Structure OTC is a trimeric protein. There are three active sites of the protein which are located at the cleft between the monomers. The carbamoyl phosphate binding domain resides on the N-terminal end of each monomer, while the C-terminal end contains the binding domain for ornithine. Both binding domains have a similar structural pattern with a central para ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Immune Response
An immune response is a physiological reaction which occurs within an organism in the context of inflammation for the purpose of defending against exogenous factors. These include a wide variety of different toxins, viruses, intra- and extracellular bacteria, protozoa, Parasitic worm, helminths, and Fungus, fungi which could cause serious problems to the health of the host organism if not cleared from the body. In addition, there are other forms of immune response. For example, harmless exogenous factors (such as pollen and food components) can trigger allergy; latex and metals are also known allergens. A transplanted tissue (for example, blood) or organ can cause graft-versus-host disease. A type of immune reactivity known as Rh disease can be observed in pregnant women. These special forms of immune response are classified as hypersensitivity. Another special form of immune response is CD4+ T cells and antitumor immunity, antitumor immunity. In general, there are two branches of ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Nanoparticle
A nanoparticle or ultrafine particle is a particle of matter 1 to 100 nanometres (nm) in diameter. The term is sometimes used for larger particles, up to 500 nm, or fibers and tubes that are less than 100 nm in only two directions. At the lowest range, metal particles smaller than 1 nm are usually called atom clusters instead. Nanoparticles are distinguished from microparticles (1-1000 μm), "fine particles" (sized between 100 and 2500 nm), and "coarse particles" (ranging from 2500 to 10,000 nm), because their smaller size drives very different physical or chemical properties, like colloidal properties and ultrafast optical effects or electric properties. Being more subject to the Brownian motion, they usually do not sediment, like colloid, colloidal particles that conversely are usually understood to range from 1 to 1000 nm. Being much smaller than the wavelengths of visible light (400-700 nm), nanoparticles cannot be seen with ordinary ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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In Vivo
Studies that are ''in vivo'' (Latin for "within the living"; often not italicized in English) are those in which the effects of various biological entities are tested on whole, living organisms or cells, usually animals, including humans, and plants, as opposed to a tissue extract or dead organism. Examples of investigations ''in vivo'' include: the pathogenesis of disease by comparing the effects of bacterial infection with the effects of purified bacterial toxins; the development of non-antibiotics, antiviral drugs, and new drugs generally; and new surgical procedures. Consequently, animal testing and clinical trials are major elements of ''in vivo'' research. ''In vivo'' testing is often employed over ''in vitro'' because it is better suited for observing the overall effects of an experiment on a living subject. In drug discovery, for example, verification of efficacy ''in vivo'' is crucial, because ''in vitro'' assays can sometimes yield misleading results with drug c ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |
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Translational Medicine
Translational medicine (often called translational science, of which it is a form) develops the clinical practice applications of the basic science aspects of the biomedical sciences; that is, it translates basic science to applied science in medical practice. It is defined by the European Society for Translational Medicine as "an interdisciplinary branch of the biomedical field supported by three main pillars: benchside, bedside, and community". The goal of translational medicine is to combine disciplines, resources, expertise, and techniques within these pillars to promote enhancements in prevention, diagnosis, and therapies. Accordingly, translational medicine is a highly interdisciplinary field, the primary goal of which is to coalesce assets of various natures within the individual pillars in order to improve the global healthcare system significantly. History Translational medicine is a rapidly growing discipline in biomedical research and aims to expedite the discovery ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   [Amazon] |