A biological target is anything within a living organism to which some other entity (like an endogenous ligand or a drug) is directed and/or binds, resulting in a change in its behavior or function. Examples of common classes of biological targets are proteins and nucleic acids.
Life is defined by the processes of metabolism and compartmentalization. This means that molecules are constantly transformed inside cells, some are being built up while other are destroyed, generating both energy and building blocks for construction. A poison is a molecule which interferes with one of these processes. A drug is a category of poison which is meant to interfere only with the metabolism of a special set of cells, which may be patient's cells (for example tumor cells) or microbes. This interference, which prevents some part of metabolism, is what gives specificity to the drug. It is the place where it interacts with some object - usually an enzyme, a receptor or an enzyme complex - needed for the cell metabolism. A drug target is a particular subset of these places, chosen because interference at this place will be both effective to prevent metabolism of the target cell, and be relatively innocuous to the host. The most common and efficient situation is that of pathogenic microbes. Indeed, in their case, the metabolism is often so different from that of man or animals that many enzyme exists which do not have counterpart in the latter, while they are essential for the microbe to multiply or to survive
a drug target is a molecular structure (chemically definable by at least a molecular mass) that will undergo a specific interaction with chemicals that we call drugs because they are administered to treat or diagnose a disease. The interaction has a connection with the clinical effect(s). This definition implies several constraints. First, the medicinal goal excludes pharmacological and biochemical tools from the present approach. Second, a major constraint is a lack of technique. Life, including disease, is dynamic, but as we do not yet directly observe the interactions of drugs and targets, and only partly notice the subsequent biochemical 'ripples' they produce; we are generally limited to 'still life' (for example, X-ray crystal structures) and to treating targets as static objects.
Target identification and validation are among the most important steps in developing a new drug. Drug target validation involves proving that either DNA, RNA, or a protein molecule is directly involved in a disease process and can be a suitable target for development of a new therapeutic drug. To qualify as ‘druggable’, a target must be accessible to the proposed drug molecule, and a measurable biological reaction must be provoked as a consequence of the drug interacting with the target. This reaction may be measured both in vitro and in vivo.
Once the target has been identified, for example by genomic or proteomic investigations, the target is then validated in functional studies. The main strategies involved in target validation are gene knockout studies and direct inhibition of the target by small molecules, peptides, antibodies or any other class of inhibitors. The knockout approach genetically inhibits the target, thereby mimicking the action of the drug and allows a drug to be modelled before it is developed.
Information about established drugs and drug-target interactions is available online via public databases such as DrugBank and the Therapeutic Targets Database. In addition, information on potentially novel drug targets can be found at CenterWatch's, Drugs in Clinical Trials Database, which identifies trends in drug development based on current clinical trials.
The Horizon cell line range offers a number of drug target Knockout cell lines ready to ship, see below. Each modified cell line is provided with a non-edited parental control to provide confidence in your results.
In addition, Horizon’s cutting edge Pathfinderscreening platforms and SAGE® in vivo models can be used in target identification and validation projects.
A drug target is a molecule in the body, usually a protein, that is intrinsically associated with a particular disease process and that could be addressed by a drug to produce a desired therapeutic effect.
A molecular target is a biological macromolecule, usually a protein that plays a crucial role in a disease pathway, and modification of which with a small molecular drug results in a desirable change in the disease condition. A target can be a bodily molecule which function abnormally (like COX2 in inflammation, EGFR in certain cancers) or a protein which is critical for the survival of a pathogenic organism (protease in HIV infection).