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.
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.
Conjugation: The addition of chemical groups to an active drug ingredient with the aim of improving drug performance.
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.
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.
miRNA: Endogenous small RNAs, structurally and 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.
Protein: Amino acid chain generated following the translation of a mRNA. Proteins have a central structural, regulatory, and catalytic function in every living cell.
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).
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 21-23 nucleotides long with a central 19-21 base-pair complementarity for the formation of dsRNA and two nucleotide overhangs at the 3' ends.
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 overhands at each of the 3 'ends'.
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.
