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

You might also read

Related Articles

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

Sort by
Same author

Expression-linked promoter selection (ELiPS) engineers short, strong ubiquitous promoters for gene therapy applications.

bioRxiv : the preprint server for biology·2026
Same author

Optogenetic WNT signaling drives germ layer self-organization in a human gastruloid model.

bioRxiv : the preprint server for biology·2026
Same author

Directed evolution of novel AAV capsids for enhanced delivery to mouse and human Schwann cells.

Molecular therapy. Nucleic acids·2026
Same author

Adeno-associated virus serotype 9 structural heterogeneity and stability characterized by charge detection mass spectrometry.

Molecular therapy. Methods & clinical development·2025
Same author

Ocular Delivery of Viral Vectors for Retinal and Choroidal Vascular Diseases: A Review.

American journal of ophthalmology·2025
Same author

Nickase fidelity drives EvolvR-mediated diversification in mammalian cells.

Nature communications·2025

Related Experiment Video

Updated: Mar 15, 2026

Methods and Tips for Intravenous Administration of Adeno-associated Virus to Rats and Evaluation of Central Nervous System Transduction
08:11

Methods and Tips for Intravenous Administration of Adeno-associated Virus to Rats and Evaluation of Central Nervous System Transduction

Published on: August 25, 2017

12.2K

Engineered AAV vectors for improved central nervous system gene delivery.

Melissa A Kotterman1, David V Schaffer2

  • 14D Molecular Therapeutics ; Emeryville, CA USA.

Neurogenesis (Austin, Tex.)
|September 9, 2016
PubMed
Summary

Researchers engineered novel adeno-associated virus (AAV) vectors using directed evolution. These enhanced AAV vectors show improved infectivity and selectivity for specific central nervous system (CNS) cell types, aiding gene therapy and neuroscience research.

Keywords:
adeno-associated virusastrocytesdirected evolutiongene deliveryneural stem cellsretinaviral engineering

More Related Videos

Widespread Transduction of Mouse Neocortical Neurons by Subarachnoid Injection of AAV2
07:40

Widespread Transduction of Mouse Neocortical Neurons by Subarachnoid Injection of AAV2

Published on: May 23, 2025

1.1K
Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
09:21

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors

Published on: January 29, 2019

38.1K

Related Experiment Videos

Last Updated: Mar 15, 2026

Methods and Tips for Intravenous Administration of Adeno-associated Virus to Rats and Evaluation of Central Nervous System Transduction
08:11

Methods and Tips for Intravenous Administration of Adeno-associated Virus to Rats and Evaluation of Central Nervous System Transduction

Published on: August 25, 2017

12.2K
Widespread Transduction of Mouse Neocortical Neurons by Subarachnoid Injection of AAV2
07:40

Widespread Transduction of Mouse Neocortical Neurons by Subarachnoid Injection of AAV2

Published on: May 23, 2025

1.1K
Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors
09:21

Production, Purification, and Quality Control for Adeno-associated Virus-based Vectors

Published on: January 29, 2019

38.1K

Area of Science:

  • Molecular Biology
  • Neuroscience
  • Gene Therapy

Background:

  • Adeno-associated viruses (AAV) are non-pathogenic parvoviruses used in gene therapy.
  • Natural AAV variants have limitations in targeted delivery to specific cell types.
  • Central nervous system (CNS) gene therapy requires vectors with enhanced infectivity and selectivity.

Purpose of the Study:

  • To engineer novel AAV vectors with improved transduction capabilities for CNS cell types.
  • To overcome delivery shortcomings of natural AAV variants.
  • To facilitate research in neurogenesis, aging, and neurodegenerative diseases.

Main Methods:

  • Directed evolution, a high-throughput molecular engineering approach.
  • Generation of novel AAV vectors with enhanced function.
  • In vitro and in vivo testing of engineered AAV vector performance.

Main Results:

  • Novel AAV vectors demonstrated enhanced infectivity in vitro.
  • Engineered vectors exhibited enhanced infectivity and selectivity in vivo for CNS cells.
  • Targeted cell types include astrocytes, neural stem cells, and retinal cells.

Conclusions:

  • Engineered AAV vectors offer improved tools for CNS gene therapy.
  • These vectors can advance understanding of neurodevelopmental and neurodegenerative processes.
  • Novel AAV vectors hold potential for treating neurological disorders.