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Related Concept Videos

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Updated: Jun 3, 2026

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun
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Published on: October 24, 2014

How does the genetic assassin select its neuronal target?

James C Stevens1, Elizabeth M C Fisher, Simon Mead

  • 1Department of Neurodegenerative Disease, University College London, Queen Square, London, WC1N 3BG, UK.

Mammalian Genome : Official Journal of the International Mammalian Genome Society
|March 5, 2011
PubMed
Summary
This summary is machine-generated.

Researchers are investigating why specific brain cells die in neurodegenerative diseases, even when causative genes are found throughout the body. Understanding this "neuronal targeting" phenomenon is key to unlocking disease mechanisms.

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Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Increasing identification of genes causative for neurodegenerative diseases.
  • Genes implicated in neurodegeneration are often ubiquitously expressed.
  • Neurodegenerative diseases exhibit specific neuronal subtype vulnerability.

Purpose of the Study:

  • To explore the phenomenon of "neuronal targeting" in neurodegenerative diseases.
  • To understand why specific neuronal subtypes are vulnerable to mutations in ubiquitously expressed genes.
  • To address the paradox of ubiquitously expressed genes causing targeted neuronal death.

Main Methods:

  • Analysis of human familial studies.
  • Investigation using animal models.
  • Dissection of molecular pathology underlying neuronal death.

Main Results:

  • Identification of numerous genes causative for human neurodegenerative diseases.
  • Observation that most identified genes are ubiquitously expressed.
  • Recognition that different mutations in the same gene can lead to varied neurodegenerative phenotypes.

Conclusions:

  • A significant gap exists in understanding the molecular mechanisms of neuronal targeting.
  • Further research is needed to explain the selective vulnerability of neuronal subtypes.
  • The phenomenon of neuronal targeting is central to understanding neurodegenerative disease pathogenesis.