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

Translational considerations for CNS gene therapy.

Deborah A Ryan1, Howard J Federoff

  • 1University of Rochester School of Medicine & Dentistry, Interdepartmental Graduate Program in Neuroscience, Rochester, New York, USA.

Expert Opinion on Biological Therapy
|February 21, 2007
PubMed
Summary

Gene therapy shows promise for challenging central nervous system (CNS) diseases. Preclinical studies explore delivery, safety, and regulation for conditions like Alzheimer's and Parkinson's disease.

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

Advantages and challenges of using allogeneic vs. autologous sources for neuronal cell replacement in Parkinson's disease: Insights from non-human primate studies.

Brain research bulletin·2025
Same author

Autologous vs heterologous cell replacement strategies for Parkinson disease and other neurologic diseases.

Handbook of clinical neurology·2024
Same author

Preface.

Handbook of clinical neurology·2024
Same author

Nigrostriatal tau pathology in parkinsonism and Parkinson's disease.

Brain : a journal of neurology·2023
Same author

E.L., a modern-day Phineas Gage: Revisiting frontal lobe injury.

Lancet regional health. Americas·2023
Same author

A retrotransposon storm marks clinical phenoconversion to late-onset Alzheimer's disease.

GeroScience·2022

Area of Science:

  • Neuroscience
  • Molecular Biology
  • Biotechnology

Background:

  • Central nervous system (CNS) diseases present significant therapeutic challenges.
  • Gene transfer is a promising approach for treating these disorders.
  • Preclinical research is essential for developing safe and effective gene therapies.

Purpose of the Study:

  • To review key considerations in developing gene therapy for CNS diseases.
  • To discuss various preclinical approaches for gene transfer.
  • To examine gene therapy strategies using Alzheimer's and Parkinson's disease models.

Main Methods:

  • Review of existing literature on gene transfer for CNS disorders.
  • Analysis of preclinical studies focusing on delivery and safety.

Related Experiment Videos

  • Case studies of Alzheimer's and Parkinson's disease gene therapy models.

    • Main Results:

    • Gene therapy development requires careful attention to delivery, expression, regulation, and safety.
    • Viral vector systems and gene products necessitate thorough toxicity and safety evaluations.
    • Preclinical models like Alzheimer's and Parkinson's disease are crucial for addressing disease-specific challenges.

    Conclusions:

    • Gene therapy holds significant potential for treating debilitating CNS diseases.
    • Addressing challenges in delivery, expression, and safety is critical for clinical translation.
    • Further preclinical research is vital for advancing gene therapy applications in neurology.