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

Gene Therapy00:59

Gene Therapy

27.2K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
27.2K
Neurogenesis and Regeneration of Nervous Tissue01:15

Neurogenesis and Regeneration of Nervous Tissue

1.5K
In the CNS, neurogenesis, the birth of new neurons from stem cells, is limited to the hippocampus in adults. In other regions of the brain and spinal cord, neurogenesis is almost non-existent due to inhibitory influences from neuroglia, especially oligodendrocytes, and the absence of growth-stimulating cues. The myelin produced by oligodendrocytes in the CNS inhibits neuronal regeneration. Furthermore, astrocytes proliferate rapidly after neuronal damage, forming scar tissue that physically...
1.5K

You might also read

Related Articles

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

Sort by
Same author

Preoperative limbic cortical thinning in mesial temporal lobe epilepsy surgical candidates is associated with pre-existing and de novo postoperative depression.

Epileptic disorders : international epilepsy journal with videotape·2026
Same author

Corrigendum.

Ophthalmology·2026
Same author

Mouse model of atypical DAT deficiency syndrome uncovers dopamine dysfunction associated with parkinsonism and ADHD.

The Journal of clinical investigation·2026
Same author

Baseline characteristics from evoke and evoke+: Two phase 3 randomized placebo-controlled trials of semaglutide in participants with early-stage symptomatic Alzheimer's disease.

Alzheimer's & dementia (New York, N. Y.)·2026
Same author

A Novel Staging Model for Uveal Melanoma: Combining Tumor Volume, Clinical Factors, and Genetic Alterations in a Danish Cohort.

Ophthalmology·2025
Same author

Incidence and tumour location of iris melanoma in Denmark: a nationwide cohort study (1943-2021).

The British journal of ophthalmology·2025

Related Experiment Video

Updated: Dec 28, 2025

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies
09:19

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies

Published on: January 4, 2015

11.1K

[Gene therapy for central nervous system disorders].

Kristoffer Nissen, Louise Klem, Rose Jeppesen

  • 1woldbye@sund.ku.dk.

Ugeskrift for Laeger
|February 14, 2020
PubMed
Summary

Gene therapy shows promise for central nervous system (CNS) diseases, with recent approvals for spinal motor atrophy and retinitis pigmentosa. Clinical trials for Parkinson

More Related Videos

GM-Free Generation of Blood-Derived Neuronal Cells
08:11

GM-Free Generation of Blood-Derived Neuronal Cells

Published on: February 13, 2021

3.3K
Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
09:44

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

Published on: May 2, 2025

551

Related Experiment Videos

Last Updated: Dec 28, 2025

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies
09:19

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies

Published on: January 4, 2015

11.1K
GM-Free Generation of Blood-Derived Neuronal Cells
08:11

GM-Free Generation of Blood-Derived Neuronal Cells

Published on: February 13, 2021

3.3K
Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
09:44

Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

Published on: May 2, 2025

551

Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Gene therapy is emerging as a viable treatment for various medical conditions.
  • Central nervous system (CNS) disorders represent a significant area of focus for gene therapy research.

Purpose of the Study:

  • This review discusses the recent advancements and potential of gene therapy for CNS diseases.
  • To highlight successful clinical applications and future prospects of gene therapy in neurology and ophthalmology.

Main Methods:

  • Review of current clinical trials and FDA/EU-approved gene therapies.
  • Analysis of gene therapy strategies targeting specific CNS diseases like Parkinson's, Huntington's, spinal motor atrophy, and retinitis pigmentosa.

Main Results:

  • Gene therapy for spinal motor atrophy has received FDA approval.
  • Successful gene therapy for retinitis pigmentosa is approved in the US and EU.
  • Promising results observed in clinical trials for Parkinson's disease (dopamine synthesis) and Huntington's disease (huntingtin synthesis).

Conclusions:

  • Gene therapy has demonstrated significant success in treating CNS diseases, particularly in ophthalmology.
  • Approved therapies for spinal motor atrophy and retinitis pigmentosa offer hope for broader CNS applications.
  • Continued research and clinical trials are expected to expand gene therapy's role in managing neurological disorders.