Target Identification Flashcards
Target identification includes which processes?
- Identifying cellular components that could be targeted for developing new drugs.
- Identifying the cellular targets (and off-target effects) of current drugs and new chemical entities (NCE).
- Identifying appropriate therapeutic doses.
- Identifying the likely efficacy of treatments.
- Identifying combination therapies that prevent or circumvent resistance.
What methods are used for target identification?
- High-content (or other) screening.
- Clinical screening.
- Molecular biochemical understanding of phenotype.
- Systems biology and systems medicine.
What can be targets for drug therapy?
Targets for drug therapy can be DNA, RNA, proteins, or membranes (to target things on the membranes or the membranes themselves doesn’t involve getting past the membrane so a wider range of compounds can be used).
What categories do most commonly used drug targets fall into?
Most targets for current drugs are enzymes or GPCRs.
Give some examples of biological drugs.
Antibodies, proteins, enzymes.
Describe the process of forward genetics when identifying new drug targets.
Forward genetics involves taking a random set of mutations (chemical mutations or viral vectors) and screening for those which give a particular behaviour/phenotype of the disease we want to know about.
Ultimately, we look at what was changed to trigger the disease (the gene that caused the mutation or the product of this), giving the target.
Why can forward genetics be difficult and time-consuming?
Because it is a random process it is difficult and time-consuming to find the behaviour/phenotype we want.
Describe the process of reverse genetics when identifying new drug targets.
Reverse genetics is more intelligent but harder; starting with a gene that may cause the illness. This is a more selective process; the specific gene is taken out or mutated and then the organism is examined.
What is genomics?
Genomics is the study of the genome.
What is the genome?
The genome is the complete set of sequences in the genetic material of an organism. It is a static blueprint, not changing much over the organism’s life.
Why doesn’t genomics show changes that have caused disease?
High definition data can be collected however disease happens regardless of this static nature, so genomics doesn’t show the changes that have caused the disease
How can genomics be used to identify targets for drug therapy?
Genomics can compare genomes from diseases and identify genes that correlate with the disease; correlating model systems with humans. The understanding of how this dysfunction changes the phenotype can then be used to identify potential target genes or gene products for drugs.
What is transcriptomics?
Transcriptomics is the study of the transcriptome.
What is the transcriptome?
This is the set of expressed genes, i.e. genes transcribed into RNA in a cell at a given point in time. This is dynamic but is mostly not functional.
What can transcriptomics be used for?
Specific details of gene expression, disease classification, identification of drug targets (mutations).
