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
Parkinson's Disease: Overview01:15

Parkinson's Disease: Overview

723
Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is...
723

You might also read

Related Articles

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

Sort by
Same author

Four ppm measurement of the antihydrogen ground-state hyperfine splitting.

Nature·2026
Same author

Topical hydrosulphide ion gels: A Paradigm shift in onychomycosis treatment.

Acta biomaterialia·2026
Same author

Be<sup>+</sup> assisted, simultaneous confinement of more than 15000 antihydrogen atoms.

Nature communications·2025
Same author

Response to correspondence from McCarthy et al. regarding maternal sepsis screening and the role of the neutrophil-to-lymphocyte ratio.

International journal of obstetric anesthesia·2025
Same author

Genomic prediction and genome-wide association study for liver abscesses in crossbred beef cattle.

Journal of animal science·2025
Same author

Understanding the impact of vitamin D supplement formulation, quality and provision to older adults in UK residential care homes.

RSC pharmaceutics·2025

Related Experiment Video

Updated: Sep 19, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

8.2K

Gene therapy in neuronopathic lysosomal storage disorders.

A Donald1, C Horgan2, M J De Castro Lopez3

  • 1Division of Neurosciences, University of Manchester, Manchester, UK; Department of Paediatric Neurology, Royal Manchester Children's Hospital, Manchester Foundation Trust, UK.

European Journal of Paediatric Neurology : EJPN : Official Journal of the European Paediatric Neurology Society
|June 4, 2025
PubMed
Summary

Gene and cell therapies offer new hope for neuronopathic lysosomal storage disorders. This review covers current approaches, challenges, and future directions for treating these rare neurodegenerative conditions.

Keywords:
Cell therapyGene therapyNeuronopathiclysosomal

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
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

8.2K

Related Experiment Videos

Last Updated: Sep 19, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
10:16

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease

Published on: December 20, 2017

8.2K
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
Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
09:54

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

8.2K

Area of Science:

  • Biochemistry
  • Genetics
  • Neurology

Background:

  • Lysosomal storage disorders (LSDs) are inherited conditions caused by enzyme deficiencies, affecting multiple body systems.
  • Neuronopathic LSDs specifically cause neurodegeneration, presenting significant challenges, particularly in pediatric populations.
  • Despite individual rarity, these disorders collectively represent a notable burden, driving therapeutic innovation.

Purpose of the Study:

  • To review the current landscape of gene and cell-based therapies for neuronopathic lysosomal storage disorders.
  • To compare adeno-associated virus (AAV) and lentiviral vector approaches in gene therapy.
  • To discuss the progress, challenges, and future directions in developing and delivering these advanced therapies.

Main Methods:

  • Review of existing literature on gene and cell therapies for LSDs.
  • Comparative analysis of AAV and lentiviral gene delivery systems.
  • Discussion of clinical application, therapeutic development, and collaborative strategies.

Main Results:

  • Gene and cell therapies are promising for treating severe pediatric LSD phenotypes.
  • AAV and lentiviral vectors show distinct advantages and disadvantages for gene therapy applications.
  • Significant progress has been made, but challenges remain in widespread patient access and therapeutic efficacy.

Conclusions:

  • Combined therapeutic strategies and increased collaboration are crucial for advancing LSD treatments.
  • Addressing delays in therapeutic development is essential for bringing innovative treatments to patients.
  • Future efforts should focus on optimizing delivery and fostering interdisciplinary partnerships to overcome therapeutic hurdles.