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level: Level 1

Questions and Answers List

level questions: Level 1

Question	Answer
initiates because there is a disease or clinical condition without suitable medical products available. - Examples are rare diseases or orphan diseases.	Drug Discovery and Development
Various processes in drug discovery:	- Target Identification and Validation - Lead Identification and Optimization - Computer Aided Drug Design - Pre-formulation Studies-include the Challenges TLC P
it is a cellular or molecular structures involved in pathology which are responsible for disease. - Receptors -gene/protein - Enzyme - Nucleic acid - Hormone - Ion channel Interacts w/ a potential drug compound>>>> clinical effect	Target
it is the process of identifying the direct molecular target i.e. protein, nucleic acid. - It is aimed to finding the efficacy target of a drug. - It is the first step in drug discovery, “druggable”	Target Identification
it is the study of genes and their function -aims to understand the structure of the genome, including the mapping genes and sequencing the DNA. - Seeks to exploit the finding from sequencing of the human and other genomes to find new drug targets.	Genomics
- it is the study of the proteome, the complete set of proteins produced by a species, using the technologies of large - scale protein separation and identification.	Proteomics
- is a branch of molecular biology that involves extensive analysis of biological data using computers, for the purpose of enhancing biological research. - It plays a key role in various stages of the drug discovery process including target identification, computer screening of chemical compounds, and pharmacogenomics.	Bioinformatics
- Is the process by which the predicted molecular target for example protein or nucleic acid - of a small molecule is verified. - Can include knockdown or overexpression of the presumed target. - During this, its association with a specific disease and its ability to regulate biological processes is tested in the body. It confirms that interactions with the target produce the desired change in the behavior of diseased cells	Target Validation
1 Is the most commonly used for RNA interference tool for including short-term silencing of protein coding genes 2 Antisense technology prevent the synthesis of specific protein	1 siRNA- Small Interfering RNA 2 Antisense Oligonucleotides
- defined as a new chemical entity that could potentially be developed into a new drug by optimizing its beneficial effects and minimizing its side effects. - a chemical compound that shows desired pharmacological activity and may initiate the development of new chemical entity, relevant compound.	LEAD COMPOUND
- use sophisticated computer modeling to predict what type of molecule may work - utilizing the 3D structure of the receptor to come up with new molecules that can bind or suit the receptor - involves structural determination of the lead target complexes using molecular modeling tools.	De Novo “scratch”
- most common way that leads are usually found - This is a key process used in drug discovery to identify hits from compound libraries that may become leads for medicinal chemistry optimization.	High-throughput Screening(HTS)
- Scientists can also genetically engineer living systems to produce disease-fighting biological molecules. - important tool in pharmaceutical drug research and development.	Biotechnology
Early Safety Test for the Lead Compound performed in living cells, in animals and via computational models.	ADME/ Tox properties
process by which a drug candidate is designed after an initial lead compound is identified, ” or altered to make them more effective and safer. Relevant aspects like affinity, selectivity, mode of action, synthesizability and ADMET-properties are typically improved Aims to maximize bonded and non-bonded interactions with the active site of selected drug targets to increase selectivity, reduce side effects and diminish toxicity, through a dedicated screening funnel of both in vitro and in vivo assays	lead optimization
➔ deliver information on the structure of the protein-ligand complex ➔ providing insights into spatial orientation of the ligand which is essential to rationally design more selective or potent ligands	X-ray Methods
An X ray method 1 used to determine nanoparticle size distributions, pore sizes, the shape of macromolecules, and characterize distances in materials 2 a non-destructive analytical technique which provides detailed information about the internal lattice of crystalline substances, including unit cell dimensions, bond-lengths, bond-angles, and details of site-ordering	. 1 Small Angle X-ray Scattering (SAXS) 2 Single-crystal X-ray diffraction
- Smaller proteins - gives structural information about binding and dynamics in physiological conditions	NMR (Nuclear Magnetic Resonance)