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 Concept Videos

Lysosomal Hydrolases01:22

Lysosomal Hydrolases

3.9K
Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...
3.9K
Lysosomes01:31

Lysosomes

19.5K
Lysosomes are membrane-enclosed spherical sacs derived from the Golgi apparatus. The most important function of the lysosome is degrading macromolecules and biological polymers that are released during membrane trafficking events such as the secretory, endocytic, autophagic, and phagocytic pathways. The degradation is carried out by several hydrolytic enzymes active in an acidic environment of the lysosomal lumen. These acid hydrolases are involved in cellular processes such as cell signaling,...
19.5K

You might also read

Related Articles

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

Sort by
Same author

Diagnostic and therapeutic applications of the glycan biomarker H3N2b in GM1 Gangliosidosis.

Molecular genetics and metabolism·2026
Same author

Efficacy and safety of efavirenz in Niemann-Pick disease type C.

Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics·2025
Same author

Sphingomyelin-induced glucocorticoid receptor alterations lead to impaired presynaptic plasticity in acid sphingomyelinase deficient neurons.

Neurobiology of disease·2025
Same author

Secondary accumulation of lyso-platelet activating factors in lysosomal storage diseases.

Molecular genetics and metabolism·2025
Same author

Conference Report: Cerebellar Development and Disease at Single-Cell Resolution.

Cerebellum (London, England)·2025
Same author

Sex-dependent upregulation in oxylipins involved in inflammation resolution in the cerebellum of Niemann-Pick disease C1 mice.

Progress in neuro-psychopharmacology & biological psychiatry·2025

Related Experiment Video

Updated: Aug 28, 2025

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons
07:43

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons

Published on: January 7, 2019

7.0K

Models to study basic and applied aspects of lysosomal storage disorders.

Ángel Gaudioso1, Teresa P Silva2, María Dolores Ledesma1

  • 1Centro Biología Molecular Severo Ochoa (CSIC-UAM), Madrid, Spain.

Advanced Drug Delivery Reviews
|September 19, 2022
PubMed
Summary

Research on lysosomal storage disorders (LSDs) is advancing with new cellular and animal models. These models improve understanding of LSDs and aid in developing effective therapies, potentially reducing animal testing.

Keywords:
Animal modelsBBBLysosomesOmicsOptogeneticsOrganoidsStorage disordersiPSCs

More Related Videos

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics
11:40

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Published on: June 23, 2022

2.6K
Quantification of Endosome and Lysosome Motilities in Cultured Neurons Using Fluorescent Probes
08:15

Quantification of Endosome and Lysosome Motilities in Cultured Neurons Using Fluorescent Probes

Published on: May 22, 2017

8.2K

Related Experiment Videos

Last Updated: Aug 28, 2025

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons
07:43

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons

Published on: January 7, 2019

7.0K
Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics
11:40

Characterization of Neuronal Lysosome Interactome with Proximity Labeling Proteomics

Published on: June 23, 2022

2.6K
Quantification of Endosome and Lysosome Motilities in Cultured Neurons Using Fluorescent Probes
08:15

Quantification of Endosome and Lysosome Motilities in Cultured Neurons Using Fluorescent Probes

Published on: May 22, 2017

8.2K

Area of Science:

  • Biomedical Research
  • Genetics and Molecular Biology
  • Cell Biology

Background:

  • Lysosomal storage disorders (LSDs) are a group of rare genetic diseases with limited treatment options and often fatal outcomes.
  • Significant challenges in LSD research include patient variability and the difficulty of studying brain conditions.
  • Lysosomes play crucial roles in cellular function, and their dysfunction underlies various diseases.

Purpose of the Study:

  • To review and summarize the experimental techniques and models currently employed in lysosomal storage disorder research.
  • To highlight advancements in both in vitro and in vivo methodologies for studying LSDs.
  • To discuss emerging technologies that may offer alternatives to traditional animal models.

Main Methods:

  • Review of established and novel in vitro techniques, including cell-based assays.
  • Analysis of various animal models developed for specific LSDs.
  • Exploration of cutting-edge technologies for disease modeling and therapeutic assessment.

Main Results:

  • Development of sophisticated cellular and animal models has significantly advanced the understanding of LSD pathology.
  • These models are instrumental in addressing challenges like patient variability and central nervous system involvement.
  • New methodologies provide insights into lysosome biology and facilitate the evaluation of therapeutic strategies.

Conclusions:

  • The reviewed techniques and models are crucial for advancing LSD research and developing novel treatments.
  • These research tools have broad implications for understanding lysosomal disorders beyond LSDs.
  • Emerging technologies show promise in refining research approaches and potentially reducing reliance on animal models.