Drug Targets/Technologies
DNA Damage
DNA damage is the result of irreversible modification of DNA nucleotides or breakage of DNA strands. Although repair systems can fix minor damage, cell death results if the damage cannot be repaired.
Energy Metabolism
Adenosine-5'-triphosphate (ATP) is the main source of energy for all cellular processes. The processes by which ATP is produced or used up are also referred to as energy metabolism.
Histones Deacetylase
Histones are proteins that bind to the DNA in the nucleus. When histones are bound tightly to the DNA transcription of genes cannot occur. Histone deacetlyases are a class of enyzmes that remove acetyl groups from lysine residues on histone proteins and allow the tighter binding of histone proteins to DNA.
Ion Channels
Ion channels allow the movement of charged particles, known as ions, across cell membranes. Ion channels function in a variety of biological pathways including the firing of neurons and muscle cells and the activation of immune cells.
Kinases
Kinases are enzymes that transfer a phosphate group from an ATP molecule to a protein substrate. This protein modification, known as phosphorylation, can have a broad range of biological effects depending on the protein that is modified.
Lipid Biosynthesis
Lipids are important for energy storage, membrane integrity, hormones, signaling, and numerous other biological functions. Lipids come in several forms including fatty acids, triglycerides, phospholipids, and cholesterol.
Natural Products
Natural products are organic molecules isolated from animals, plants, or microbes that can be used to treat human disease.
Nucleic Acid Synthesis
Nucleic acids, including deoxynucleic acid (DNA) and ribonucleic acid (RNA), store genetic information for living organisms. The production and regulation of these biological macromolecules are essential for survival and replication of organisms. Therefore, enzymes involved in these processes are attractive therapeutic targets for a variety of diseases.
Proteases
Proteases are enzymes that are able to cleave or degrade other proteins. The functional and regulatory capacities of proteases vary greatly and include, but are not limited to, blood clotting, extracellular matrix degradation, activation of apoptosis, protein degradation, and viral protein processing.
Protein Synthesis
Protein synthesis is the process whereby amino acids are linked together to form proteins using mRNA as a template.
Vaccine Technology
DNA Vaccines
DNA vaccines are circular pieces of DNA, known as plasmids, that contain the sequence(s) for one or more protein antigens and replicate autonomously from the cell chromosome.. When the DNA plasmid is introduced into human cells, the human cells express the protein encoded on the plasmid, subsequently stimulating an immune response against the encoded antigen.
Inactivated Vaccines
Inactivated vaccines use killed organisms to protect against subsequent infection with live organisms that cause disease.
Live Attenuated Vaccines
Live attenuated vaccines are created by reducing the ability of an infectious organism to cause disease without killing the organism. Vaccination with the live but weakened organism generates an immune response that protects the vaccinated person against severe disease.
Polysaccharide Protein Conjugate Vaccines
Polysaccharide protein conjugate vaccines consist of polysaccharides, generally from the surface coat of bacteria, linked to protein carriers. The combination of the polysaccharide and protein carrier induces an immune response against bacteria displaying the vaccine polysaccharide on their surface, thus preventing disease.
Recombinant or Purified Protein Vaccines
Recombinant or purified protein vaccines consist of protein antigens that have either been produced in a heterologous expression system (e.g., bacteria or yeast) or purified from large amounts of the pathogenic organism. The vaccinated person produces antibodies to the protein antigen, thus protecting him/her from disease.
Viral Vector Vaccines
Viral vector vaccines use live viruses to carry DNA into human cells. The DNA contained in the virus encodes antigens that, once expressed in the infected human cells, elicit an immune response.
Diagnostic Technologies
Cell-based Diagnostics
Cell-based diagnostics rely on the evaluation of whole cells to detect the presence of disease. Cell-based assays are the gold standard for detection of the majority of infectious organisms. However, cell-based diagnostics generally require sophisticated laboratory facilities and highly skilled personnel that are not practical for use in low resources settings.
Immunoassays
An immunoassay is a diagnostic test that uses the reaction of an antibody to an antigen to diagnose a disease. When a patient is infected with a pathogen, the pathogen can be detected directly, by measuring the presence of antigens. Alternatively, the host immune response to a pathogen can be detected by measuring the presence of antibodies.
Nucleic Acid-based Diagnostics
Nucleic acid-based diagnostics detect the presence of a pathogen either by directly detecting the presence of DNA or RNA nucleic acids in the host or by first amplifying the pathogen DNA or RNA. Nucleic acid-based diagnostics are a standard central laboratory technique, although simplified nucleic acid-based diagnostics that may be useful in resource-poor settings are emerging.
Rapid Diagnostic Test (RDT)
Rapid diagnostic tests (RDTs) are diagnostic assays designed for use at the point-of-care (POC), and can be adapted for use in low-resource settings. An RDT is low-cost, simple to operate and read, sensitive, specific, stable at high temperatures, and works in a short period time. RDTs are already in use for several neglected diseases.