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Related Concept Videos

Human Genetics01:28

Human Genetics

1.6K
Human genetics provides a profound framework for understanding the interplay between genetic predispositions and human psychology. At the heart of this discipline lies the study of how genes influence physical traits, behaviors, and susceptibility to diseases. Each person carries a unique genetic code that subtly or significantly shapes their psychological and behavioral landscape.
The complex relationship between genetics and psychology is observable through common biological components such...
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Translation01:31

Translation

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Lesson: Translation
Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of...
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Translation01:31

Translation

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Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
Translation Produces the Building Blocks of Life
Proteins are...
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Targets for Drug Action: Overview01:26

Targets for Drug Action: Overview

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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...
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Initiation of Translation02:33

Initiation of Translation

39.1K
Initiating translation is complex because it involves multiple molecules. Initiator tRNA, ribosomal subunits, and eukaryotic initiation factors (eIFs) are all required to assemble on the initiation codon of mRNA. This process consists of several steps that are mediated by different eIFs.
First, the initiator tRNA must be selected from the pool of elongator tRNAs by eukaryotic initiation factor 2 (eIF2). The initiator tRNA (Met-tRNAi) has conserved sequence elements including modified bases at...
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Termination of Translation01:44

Termination of Translation

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The large ribosomal subunit has several important structures essential to translation. These include the peptidyl transferase center (PTC) - which is the site where the peptide bond is formed - and a large, internal, water-filled tube through which the nascent polypeptide moves. This latter structure is called the Peptide Exit Tunnel, and it begins at the PTC and spans the body of the large ribosomal subunit. During translation, as the nascent polypeptide chain is synthesized, it passes through...
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Related Experiment Video

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Author Spotlight: Targeted Microinjection and Electroporation of Primate Cerebral Organoids for Genetic Modification
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Translating Human Genetics into Novel Drug Targets.

Karol Estrada1,

  • 1Translational Genome Sciences, Biogen, Cambridge, MA, USA. karol.estrada@biogen.com.

Methods in Molecular Biology (Clifton, N.J.)
|June 8, 2018
PubMed
Summary

The human genome project identifies genetic causes of diseases, but translating these findings into new drug therapies faces significant challenges. This work explores opportunities and hurdles in using human genetics for novel drug target identification and validation.

Keywords:
Drug targetsHuman geneticsTranslation genomics

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Area of Science:

  • Genetics
  • Pharmacology
  • Drug Discovery

Background:

  • The Human Genome Project aimed to identify genetic causes of diseases for novel therapy development.
  • Hundreds of genetic loci linked to diseases have been identified in the past decade.
  • Translating genetic findings into effective treatments remains a complex challenge.

Purpose of the Study:

  • To describe common challenges in translating human genetic discoveries into drug therapies.
  • To highlight opportunities for utilizing human genetics in identifying and validating drug targets.
  • To address overlooked aspects of gene mapping in therapeutic development.

Main Methods:

  • Review of human genetics findings and their application in drug discovery.
  • Analysis of challenges in the translation of genetic loci to therapeutic targets.
  • Exploration of strategies for target validation using genetic data.

Main Results:

  • Numerous genetic loci associated with human traits and diseases have been identified.
  • Significant challenges exist in converting genetic discoveries into viable drug therapies.
  • Opportunities lie in leveraging human genetics for precise drug target identification.

Conclusions:

  • Human genetics offers a powerful avenue for identifying novel drug targets for diseases with high unmet need.
  • Overcoming translational challenges is crucial for realizing the full therapeutic potential of genomic research.
  • Strategic approaches are needed to validate genetic targets and develop effective treatments.