Glossary

Antisense technology: Oligonucleotides designed to bind target RNAs (e.g. mRNA, microRNA, etc.) based on sequence complementarity in order to block their natural function. Antisense technology can also describe a general approach that includes RNAi and siRNAs.
Monoclonal antibody: A single type of purified antibody derived from a clone of antibody-producing cells that can be generated in the laboratory and used for drug development where a therapeutic benefit is expected from the binding of the antibody to a protein target.
Bioinformatics: Computational biology concerned with the management and analysis of biological data.
Canonical siRNA: Two strands of RNA, each 21 nucleotides long with a central region of complementarity that is 19 base-pairs long for the formation of dsRNA and two nucleotide overhangs at each of the 3' ends.
Conjugation: The addition of chemical groups to an active drug ingredient with the aim of improving drug performance.
Central Nervous System (CNS): This system consists of the brain and spinal cord, where many bodily functions are controlled, many sensations are processed and signals are sent to different parts of the body. Functions affected by the CNS include muscle control, eyesight, breathing and memory. The CNS is distinguished from the peripheral nervous system, which involves the actual nerves to and from the muscles and other body parts.
Dicer: Central enzyme in the natural RNAi pathway that generates the active small RNAs by cleaving dsRNA precursors.
DNA: Deoxyribonucleic acid; genetic material consisting of any sequence of deoxyribonucleotides containing bases of adenine (A), thymine (T), guanine (G), and cytosine (C).
dsRNA: Double-stranded RNA; matching of a chain of ribonucleic acid, RNA, by a complementary strand of RNA. In this conformation, an adenine nucleotide present in one strand bonds, or base-pairs, with a uracil nucleotide on the complementary strand, and a guanine nucleotide to a cytosine.
High Density Lipoprotein (HDL): Lipoprotein particle in the blood. HDL is known as "good" cholesterol because it deposits cholesterol in the liver, where it is excreted by the body. High HDL is thought to protect against coronary artery disease.
Hypercholesterolemia: a condition characterized by very high levels of cholesterol in the blood. The body needs cholesterol to build cell membranes, make certain hormones, and produce compounds that aid in fat digestion. Too much cholesterol, however, increases a person's risk of developing heart disease.
Investigational New Drug (IND): An application containing laboratory (pre-clinical) study results of a drug candidate is submitted to the FDA to request permission to conduct studies in humans.
Gene: Basic genetic unit; DNA sequence that carries all the information for the functional production of a protein or non-coding RNA.
Genome: The entire DNA sequence of an organism.
Kinesin spindle protein (KSP): Also known as "eglin 5" or "Eg5", is a protein required for cell division that, when inhibited, leads to cell arrest and cell death in dividing cells.
Low Density Lipoprotein (LDL): A lipoprotein particle in the blood responsible for depositing cholesterol into the lining of the artery. Known as "bad" cholesterol because high LDL is linked to coronary artery disease.
microRNA: Endogenous small RNAs, functionally related to siRNAs, involved in negatively regulating the expression of a large number of genes.
mRNA: Messenger RNA functioning as the template in protein translation. They are generated by the transcription of protein-coding genes.
Pharmacokinetics: Study on the absorption, distribution, metabolism, and elimination of a drug in the body over time.
Phase I clinical trial: The initial set of drug studies in humans, which are generally designed to evaluate the safety of a new drug in a small number of patients or normal volunteers.
Phase II: Controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication or indications in patients with the disease or condition under study and to determine the common short-term side effects and risks.
Phase III clinical trial: These studies are the definitive trails conducted to demonstrate the safety and effectiveness of a new drug for the diagnosis or treatment of a disease.
Protein: Amino acid chain generated following the translation of a mRNA. Proteins have a central structural, regulatory, and catalytic function in every living cell.
PSCK9: Proprotein convertase subtilisin/kexin type 9. The PCSK9 gene provides instructions for making a protein that helps regulate the amount of cholesterol in the bloodstream. Hypercholesterolemia is caused by mutations in the PCSK9 gene.
Pre-clinical: The testing of experimental drugs in the test tube or in animals before trials in humans may be carried out.
RiSC: RNA-induced silencing complex which when guided by an incorporated small RNA cleaves complementary target mRNA during RNAi.
RNA: Ribonucleic acid; genetic information consisting of any sequence of ribonucleotides containing bases of adenine (A), uracil (U), guanine (G), and cytosine (C).
RNAa: RNA activation, double-stranded RNAs that target promoter regions in chromosomal DNA resulting in transcriptional activation of genes. The transcriptional activation, or up-regulation, of genes results in an increase in mRNA and protein production.
RNAi: RNA interference, a natural cellular process where small interfering RNAs or microRNAs are used to control the normal expression of genes or are used to pharmacologically target disease-causing genes.
Respiratory syncytial virus (RSV): a highly contagious virus that causes infections in both the upper and lower respiratory tract. RSV infects nearly every child at least once by the age of two years and is a major cause of hospitalization due to respiratory infection in children and people with compromised immune systems, and others.
shRNA: Small hairpin RNA expressed from a DNA template and processed into small RNAs to guide RNAi-mediated target mRNA degradation.
siRNA: Small interfering RNA. Optimally, two strands of RNA, each 19-25 nucleotides long with a central region of complementarity for the formation of dsRNA and optionally nucleotide overhangs at one or both of the the 3' ends. siRNAs are the molecules that mediate RNAi. They are incorporated into RISC and used to form complementary pairing with a target mRNA, resulting in its enzymatic cleavage.
Small molecule: A small chemical, usually drug that due to its small size may be absorbed through the gut. Term often used in contrast to "large" protein therapeutics which have to be injected to bypass the gut.
TTR amyloidosis: A hereditary, systemic disease caused by a mutation in the transthyretin (TTR) gene. The mutation causes abnormal amyloid proteins to accumulate in and damage body organs and tissue such as the peripheral nerves and heart, resulting in neuropathic pain, autonomic neuropathy, and cardiomyopathy.
Vascular endothelial growth factor (VEGF): A protein that is secreted by oxygen-deprived cells, such as cancerous cells. VEGF stimulates new blood vessel formation, or angiogenesis, by binding to specific receptors on nearby blood vessels, encouraging new blood vessels to form.