Acetylation
A reaction that introduces an acetyl function group into a molecule, i.e. co-translational or post-translational modification of a protein.
Agonist
A chemical that binds to a receptor of a cell and triggers a response by that cell. Agonists often mimic the action of a naturally occurring substance.
Allele
One of a number of different forms of a gene. Each person inherits two alleles for each gene, one allele from each parent. These alleles may be the same or may be different from one another.
Amino acid
One of the 20 building blocks of protein. The sequence of amino acids in a protein and, hence, the function of that protein are determined by the genetic code in the DNA.
Aminoglycoside
A molecule or portion of a molecule composed of amino-modified sugars.
Assay
A chemical or biological test in which the activity of a biological target molecule (often a protein) is measured in the presence of other substances (often chemicals).
Astrocyte
Star-shaped glial cell.
Autonomic nervous system
The portion of the nervous system that regulates involuntary body functions, including those of the heart and intestines. Controls blood flow, digestion and temperature regulation.
BDNF
Brain-derived neurotrophic factor – a protein encoded by the BDNF gene, a growth protein that induces the survival, development and function of neurons.
Blood-brain barrier
A protective barrier separating circulating blood from cerebrospinal fluid.
Cell
Basic functional unit of life in any living organism.
Chromatin
The combination of DNA, histone and other proteins that make up chromosomes.
Chromosome
An organized structure of DNA and protein that is found in cells; it is a single piece of coiled DNA containing many genes.
Clinical trial
Testing the safety and effectiveness of any potential therapy on live patients.
Compounds
A collection of distinct defined and characterized molecules or mixtures thereof.
Compound libraries
Collections of compounds that provide a variety of chemically diverse structures that can be used to identify structure types that have affinity with pharmacological targets.
Cytoplasm
A thick, clear liquid residing between the cell membrane holding organelles.
DNA
Deoxyribonucleic acid; contains the genetic instructions used in the development and functioning of all known living organisms with the exception of some viruses.
Drug discovery
The process by which drugs are discovered and/or designed.
Dystonia
Abnormal and awkward posture or sustained movements of a hand, foot, or other part of the body; may be accompanied by rigidity and twisting.
Endogenous
From inside the body.
Enzyme
A protein that catalyses (i.e. increases or decreases the rates of) chemical reactions.
Epigenetics
Over and above genetics – the study of inherited changes in phenotype or gene expression caused by mechanisms other than changes in the underlying DNA sequence.
Excitatory (type of neurotransmitter)
The most important receptors increase the probability that the target cell will fire an action potential.
Exogenous
From outside of the body.
Expression
The actual production of the protein which the gene encodes.
GABA
The chief inhibitory neurotransmitter in the central nervous system.
Gene
A portion of a chromosome (DNA) that contains the hereditary information necessary for the production of a protein.
Gene transcription
Encodes at least 1 gene (produces mRNA) from a stretch of DNA.
Gene translation
mRNA (messenger RNA) is decoded to produce an amino acid chain, which will later fold into an active protein.
Genotype
The genetic makeup of an organism, as distinguished from its physical appearance (the phenotype).
High throughput screen
High-throughput screening (HTS) aims to rapidly assess the activity of a large number of compounds or extracts on a given target.
Histones
Strongly alkaline proteins, which package and order the DNA into structural units called nucleosomes. They are the chief protein components of chromatin, act as spools around which DNA winds, and play a role in gene regulation.
Hits
A compound that shows activity in a primary screen.
Intracerebroventricular (ICV)
A method of administering fluid directly into the ventricles by means of surgically-implanted osmotic minipumps that deliver solution at a constant rate.
In vitro
Testing in a petri dish.
In vivo.
The opposite of in vitro (outside the body or in the laboratory). In this case, experiments that will take place in living organisms.
Inhibitory (type of neurotransmitter)
The most important receptors will have an inhibitory effect.
Knockout
A knockout mouse is a genetically engineered mouse one or more of whose genes have been made inoperable.
Lead
A compound with a confirmed activity profile that warrants development.
Ligand
A molecule that attaches (or binds) to a receptor.
Long term potentiation (LTP)
A long lasting enhancement in signal transmission between two neurons that results from stimulating them synchronously. It’s one of several phenomena underlying synaptic plasticity. LTP is widely considered to b one of the major cellular mechanisms that underlies learning and memory.
Metabolism
Set of chemical reactions that happen in living organisms to maintain life.
Methylation
The addition of a methyl group, or the substitution of an atom or group by a methyl group. Makes protein active or not.
Microglia
Glial cells residing in the brain but that originally come from hematopoietic stem cells in blood or bone marrow. Are involved in immune functions.
Missense mutation
Amino acid changed to produce an altered amino acid in the protein product.
Mitochondrion
A membrane-enclosed organelle found in most cells with complex structures.
Molecule
Group of at least two atoms.
mRNA
Messenger RNA. All organisms use mRNA to carry the genetic information that directs the synthesis of proteins.
Neuron
Nerve cell – an electrically excitable cell that processes and transmits information by electrical and chemical signaling.
Neuroplasticity
Ability of the brain to change as a result of experience.
Neurotransmitter
Chemical produced by the body that transmits signals from a neuron to a target cell across a synapse. It can come into existence from the transformation of an amino acid, for example.
Nonsense mutation
Amino acid is changed to a stop codon which prematurely truncates a protein.
Nucleotide
Molecules that, when joined together, make up the structural units of RNA and DNA.
Oligodendrocytes
A type of glia cell.
Pathogenesis
Step by step development of a disease. The mechanism by which a disease is caused.
Phenotype
The observable traits or characteristics of an organism.
Phosphate
A chemical compound.
Plasma membrane
Surrounds the protoplasm of a cell.
Protein
A large biomolecule composed of one or more chains of amino acids in a specific order. Proteins are required for the structure, function, and regulation of cells, tissues, and organs.
Receptor
A protein molecule, embedded in the plasma membrane of a cell, or the cytoplasm of a cell, to which one or more signaling molecules may attach (or bind). A molecule that attaches (or binds) is called a ligand.
RNA
Ribonucleic acid (RNA) is one of the three major macromolecules (along with DNA and proteins) that are essential for all forms of life. The sequence of nucleotides allows RNA to encode genetic information.
Screen
Testing large numbers of compounds in order to identify those with particular characteristics.
Somatic cell
Any cell in the body, except for germline cells (spermatozoa and ova).
Synapse
Junction box that allows electric or chemical signal to be transmitted from a neuron to another cell.
Synaptic plasticity
The ability of chemical synapses to change their strength.
Vector
Organism used to transmit the corrective gene.
Wildtype
The type or form of an organism or gene that occurs most frequently in nature. Often refers to how organisms or genes are found naturally, in the wild, in comparison to mutated versions.
X chromosome
The sex chromosome that is present in both sexes: singly in males and doubly in females.
X-inactivation, XCI, XI
In normal females with two X chromosomes, the genes on one or the other of them are silenced to avoid duplication, or over expression. Typically, each X contributes roughly half the active genetic information.

