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

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Human Genetics01:28

Human Genetics

Human genetics provides a profound framework for understanding the interplay between genetic predispositions and human psychology. At the heart of this discipline lies the study of how genes influence physical traits, behaviors, and susceptibility to diseases. Each person carries a unique genetic code that subtly or significantly shapes their psychological and behavioral landscape.
The complex relationship between genetics and psychology is observable through common biological components such...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
Neurogenesis and Regeneration of Nervous Tissue01:15

Neurogenesis and Regeneration of Nervous Tissue

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...

You might also read

Related Articles

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

Sort by
Same author

Recent progress and considerations for AAV gene therapies targeting the central nervous system.

Journal of neurodevelopmental disorders·2018
Same author

Insights into the Pathogenesis and Treatment of Krabbe Disease.

Pediatric endocrinology reviews : PER·2016
Same author

Viral expression cassette elements to enhance transgene target specificity and expression in gene therapy.

Discovery medicine·2015
Same author

Combination therapy utilizing shRNA knockdown and an optimized resistant transgene for rescue of diseases caused by misfolded proteins.

Proceedings of the National Academy of Sciences of the United States of America·2011

Related Experiment Video

Updated: May 20, 2026

Rapid Detection of Neurodevelopmental Phenotypes in Human Neural Precursor Cells (NPCs)
10:47

Rapid Detection of Neurodevelopmental Phenotypes in Human Neural Precursor Cells (NPCs)

Published on: March 2, 2018

Gene therapy and neurodevelopmental disorders.

Steven James Gray1

  • 1Gene Therapy Center, University of North Carolina at Chapel Hill, 7109 Thurston Bowles, 104 Manning Drive, Chapel Hill, NC 27599-7352, USA. graysj@email.unc.edu

Neuropharmacology
|July 4, 2012
PubMed
Summary
This summary is machine-generated.

Gene therapy shows promise for neurological disorders, particularly with advances in central nervous system (CNS) gene delivery using adeno-associated virus (AAV) vectors. Challenges remain for neurodevelopmental disorders like Rett Syndrome and autism.

More Related Videos

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

Related Experiment Videos

Last Updated: May 20, 2026

Rapid Detection of Neurodevelopmental Phenotypes in Human Neural Precursor Cells (NPCs)
10:47

Rapid Detection of Neurodevelopmental Phenotypes in Human Neural Precursor Cells (NPCs)

Published on: March 2, 2018

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

Area of Science:

  • Neurology
  • Genetics
  • Biotechnology

Background:

  • Gene therapy is emerging as a viable treatment for neurological disorders.
  • Central nervous system (CNS) gene delivery has seen significant advancements.
  • Lysosomal storage diseases are nearing gene therapy solutions.

Purpose of the Study:

  • To review the current state of adeno-associated virus (AAV) vector design and delivery for CNS gene therapy.
  • To discuss gene therapy applications for neurodevelopmental disorders.
  • To highlight remaining challenges in the field.

Main Methods:

  • Review of current literature on AAV vector technology for CNS gene delivery.
  • Analysis of gene therapy studies targeting neurodevelopmental disorders.
  • Discussion of challenges and future directions in AAV-mediated gene therapy.

Main Results:

  • Adeno-associated virus (AAV) is the most common vector for CNS gene delivery.
  • Progress has been made in treating some neurological disorders, but significant hurdles exist for others.
  • Specific examples of gene therapy studies for neurodevelopmental disorders are presented.

Conclusions:

  • AAV vectors are crucial for CNS gene therapy development.
  • While promising, gene therapy for neurodevelopmental disorders like Rett Syndrome, Fragile X, and autism still requires overcoming substantial obstacles.
  • Continued research in AAV vector design and delivery is essential for advancing neurological disorder treatments.