Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Video

Updated: May 29, 2026

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis (ALS)
12:35

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis (ALS)

Published on: March 17, 2012

Animal models for Gaucher disease research.

Tamar Farfel-Becker1, Einat B Vitner, Anthony H Futerman

  • 1Department of Biological Chemistry, Weizmann Institute of Science, Rehovot 76100, Israel.

Disease Models & Mechanisms
|September 29, 2011
PubMed
Summary

Developing accurate animal models for Gaucher disease (GD), a lysosomal storage disorder, remains challenging. This review examines existing models and proposes criteria for creating better ones to study GD pathology and treatments.

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

It's time to rethink academic innovation: why technology transfer offices can't do it alone.

Nature biotechnology·2026
Same author

Discovery of a potential CERS2 inhibitor: hit compound identification via structure-based virtual screening and molecular dynamics simulations.

Molecular diversity·2026
Same author

Nucleolin perturbation alters membrane lipid homeostasis.

Molecular omics·2025
Same author

Ultrafast Measurement of Circulating Ceramides in Human Cohorts.

Analytical chemistry·2025
Same author

In Vivo Accumulation of Regulatory T Cells Using Eliglustat-Loaded Cryogels.

Advanced healthcare materials·2025
Same author

Deep sphingolipidomic and metabolomic analyses of ceramide synthase 2 null mice reveal complex pathway-specific effects.

Journal of lipid research·2025

Area of Science:

  • Biochemistry
  • Genetics
  • Animal Models

Background:

  • Gaucher disease (GD) is the most common lysosomal storage disorder (LSD).
  • It results from defective glucocerebrosidase activity, encoded by the GBA gene.
  • Creating animal models that fully represent GD's clinical subtypes has been difficult.

Purpose of the Study:

  • To review the utility of current animal models for Gaucher disease research.
  • To assess their role in understanding GD pathological pathways.
  • To evaluate their effectiveness in testing novel therapeutic strategies.

Main Methods:

  • Review of existing literature on Gaucher disease animal models.
  • Analysis of the successes and limitations of various model systems.

Related Experiment Videos

Last Updated: May 29, 2026

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis (ALS)
12:35

Clinical Testing and Spinal Cord Removal in a Mouse Model for Amyotrophic Lateral Sclerosis (ALS)

Published on: March 17, 2012

  • Identification of criteria for improved model generation.

    • Main Results:

    • Early mouse models for GD exhibited severe limitations, including early mortality and lack of disease correlation.
    • Conditional knockout mice now show promise in mimicking some human GD features.
    • Current models offer insights into GD pathology and treatment efficacy.

    Conclusions:

    • Accurate animal models are crucial for advancing Gaucher disease research.
    • Further refinement is needed to develop models that fully recapitulate GD subtypes.
    • Establishing clear criteria will guide the creation of more suitable GD models.