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 Videos

Gene therapy for lysosomal storage disorders.

N S Yew1, S H Cheng

  • 1Genzyme Corporation, Framingham, MA 01701-9322, USA. nelson.yew@genzyme.com

Current Opinion in Molecular Therapeutics
|August 30, 2001
PubMed
Summary

Gene therapy offers a promising new avenue for treating severe lysosomal storage diseases, especially those affecting the nervous system, with recent advances showing significant potential for these currently untreatable genetic disorders.

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

Sequencing of Pleural Fluid and Plasma for Tuberculous Pleuritis.

NEJM evidence·2026
Same author

Unique Berry phase in one-dimensional topological photonic systems.

Optics letters·2025
Same author

Improved risk stratification of nasopharyngeal cancer by targeted sequencing of Epstein-Barr virus DNA in post-treatment plasma.

Annals of oncology : official journal of the European Society for Medical Oncology·2022
Same author

[Clinical analysis of 15 cases with myeloperoxidase antineutrophil cytoplasmic antibody associated hypertrophic pachymeningitis].

Zhonghua nei ke za zhi·2022
Same author

[Clinical characteristics of patients with antisynthetase syndrome and positive anti-Ro52 antibody].

Zhonghua nei ke za zhi·2022
Same author

[The role of Artificial intelligent-based FFR<sub>CT</sub> in assessing the hemodynamic relevance of deep myocardial bridge of the left anterior descending coronary artery].

Zhonghua yi xue za zhi·2021

Area of Science:

  • Biochemistry
  • Genetics
  • Neurology

Background:

  • Lysosomal storage diseases stem from deficiencies in lysosomal enzymes.
  • Most lysosomal storage diseases lack effective therapeutic options.
  • Neurological manifestations are common and severe in many lysosomal storage diseases.

Purpose of the Study:

  • To review recent advancements in gene therapy for lysosomal storage diseases.
  • To highlight gene therapy as a potential treatment for neurological manifestations.
  • To provide an overview of a large and diverse group of genetic disorders.

Main Methods:

  • Literature review of recent gene therapy research.
  • Analysis of therapeutic strategies for lysosomal storage diseases.
  • Focus on studies addressing neurological aspects.

Main Results:

  • Gene therapy is emerging as a viable treatment approach.
  • Significant progress has been made in developing gene-based therapies.
  • Gene therapy shows particular promise for neurological lysosomal storage diseases.

Conclusions:

  • Gene therapy represents a hopeful frontier for treating lysosomal storage diseases.
  • Further research and development are crucial for clinical application.
  • Gene therapy may offer a transformative treatment for patients with limited options.

Related Experiment Videos