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Related Concept Videos

Drug Discovery: Overview01:26

Drug Discovery: Overview

Drug discovery is a multifaceted process involving extensive screening, testing, and optimization of lead compounds to identify potential new drugs for therapeutic use. It combines several approaches, including screening large numbers of natural products, chemical modification of known active molecules, identification of new drug targets, and rational design based on biological mechanisms and drug-receptor structure. These approaches are carried out in both academic research laboratories and...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Structure-Activity Relationships and Drug Design01:28

Structure-Activity Relationships and Drug Design

Drug design is a dynamic field that involves discovering and developing new medications based on specific biological targets. This process heavily relies on structure-activity relationships (SAR) and quantitative structure-activity relationships (QSAR) to guide the design and optimization of efficient drugs.
SAR studies the intricate relationship between a drug's chemical structure and biological activity. It focuses on understanding how modifications to a drug's structure can influence its...
Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
Protein-protein Interfaces02:04

Protein-protein Interfaces

Many proteins form complexes to carry out their functions, making protein-protein interactions (PPIs) essential for an organism's survival. Most PPIs are stabilized by numerous weak noncovalent chemical forces. The physical shape of the interfaces determines the way two proteins interact. Many globular proteins have closely-matching shapes on their surfaces, which form a large number of weak bonds. Additionally, many PPIs occur between two helices or between a surface cleft and a polypeptide...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...

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Protein Target Prediction and Validation of Small Molecule Compound
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Published on: February 23, 2024

Main approaches to target discovery and validation.

Mouldy Sioud1

  • 1Department of Immnology, Institute for Cancer Research, The Norwegian Radium Hospital, University of Oslo, Oslo, Norway.

Methods in Molecular Biology (Clifton, N.J.)
|December 19, 2006
PubMed
Summary

Identifying disease-causing genes is crucial for drug discovery. Phenotype-oriented approaches and reverse genetics in mice help validate these targets for therapeutic development and vaccine design.

Area of Science:

  • Biomedical Research
  • Genetics
  • Pharmacology

Background:

  • Drug discovery relies on identifying and validating disease-causing genes.
  • Genomics, proteomics, and bioinformatics reveal disease pathways and potential therapeutic targets.
  • Phenotype-oriented target identification directly links genetic changes to disease manifestations.

Purpose of the Study:

  • To outline the essential steps in identifying and validating disease-causing genes for drug development.
  • To highlight the importance of phenotype-oriented approaches and reverse genetics.
  • To emphasize the role of target validation in creating effective therapeutics and vaccines.

Main Methods:

  • Utilizing genomics, proteomics, and bioinformatics to identify potential targets.

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  • Employing phenotype-oriented target identification for direct genotype-phenotype correlation.
  • Leveraging mouse reverse genetics (e.g., knockout phenotypes) for in vivo validation.
  • Exploring strategies for gene expression modulation (antibodies, antisense oligonucleotides, etc.).

    • Main Results:

    • Phenotype-oriented identification increases the likelihood of finding causal disease genes.
    • Mouse reverse genetics provides a powerful method for validating gene function in mammalian physiology.
    • Target discovery and validation are key to developing new drugs and vaccines.

    Conclusions:

    • Accurate identification and validation of disease targets are fundamental to advancing drug discovery and development.
    • Integrating various approaches, including phenotype-oriented methods and reverse genetics, strengthens the validation process.
    • Successful target validation paves the way for novel therapeutic interventions and improved vaccine strategies.