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

ApoE lipoproteins in the central nervous system under homeostasis and role in Alzheimer's disease and related disorders.

Molecular neurodegeneration·2026
Same author

Draft genome sequences of three non-O1/non-O139 <i>Vibrio cholerae</i> isolates from Lake Wonderwood near Seminole Beach, Florida.

Microbiology resource announcements·2026
Same author

Data resource profile: PharmaNet, the database for prescription drug dispensing in British Columbia, Canada.

International journal of population data science·2026
Same author

Dual-transcriptomic analysis of human nasal transcriptome and microbiome reveals host-bacteria associations in symptomatic respiratory infection.

BMC genomics·2026
Same author

Multi-omic phenotyping of MAPT V337M neurons reveals early changes in axonogenesis and tau phosphorylation.

NPJ dementia·2026
Same author

Outcomes of peroral endoscopic myotomy in patients with cirrhosis: a multicenter experience.

Gastrointestinal endoscopy·2026

Related Experiment Video

Updated: Mar 19, 2026

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

Microglia-specific targeting by novel capsid-modified AAV6 vectors.

Awilda M Rosario1, Pedro E Cruz1, Carolina Ceballos-Diaz1

  • 1Department of Neuroscience, Center for Translational Research in Neurodegenerative Disease, University of Florida , Gainesville, Florida, USA.

Molecular Therapy. Methods & Clinical Development
|June 17, 2016
PubMed
Summary

Researchers developed novel recombinant adeno-associated virus (rAAV) capsid variants to target microglia, the brain's immune cells. This breakthrough enables microglia-specific gene expression for studying central nervous system diseases.

More Related Videos

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
09:20

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Published on: October 18, 2022

5.4K
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 19, 2026

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
Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants
09:20

Isolation of Next-Generation Gene Therapy Vectors through Engineering, Barcoding, and Screening of Adeno-Associated Virus AAV Capsid Variants

Published on: October 18, 2022

5.4K
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:

  • Neuroscience
  • Immunology
  • Gene Therapy

Background:

  • Recombinant adeno-associated viruses (rAAV) are key tools for gene therapy in the central nervous system.
  • Microglia, the brain's resident immune cells, are typically resistant to rAAV transduction.
  • Targeting microglia is crucial for understanding and treating neuroinflammatory and neurodegenerative diseases.

Purpose of the Study:

  • To identify adeno-associated virus (AAV) capsids capable of microglia-specific gene delivery.
  • To develop novel rAAV vectors for functional studies and gene therapy targeting microglia.
  • To investigate the potential of engineered AAV capsids for modulating microglial activity.

Main Methods:

  • Screening of common AAV serotypes (AAV1-9, rh10) for microglial transduction.
  • Testing modified rAAV6 capsid variants, including a Y731F/Y705F/T492V mutant, on primary mouse microglia cultures.
  • In vivo delivery of rAAV constructs with microglia-specific promoters (F4/80, CD68) in mouse brains.
  • Assessment of gene expression and downstream effects like astrogliosis.

Main Results:

  • Standard AAV serotypes showed limited transduction of primary mouse microglia.
  • Engineered rAAV6 capsid variants, particularly the Y731F/Y705F/T492V mutant, efficiently and selectively transduced microglia in vitro.
  • In vivo studies demonstrated microglia-specific expression of reporter genes and interleukin-6.
  • CD68 promoter-driven IL-6 expression induced astrogliosis, indicating functional modulation of the innate immune system.

Conclusions:

  • Novel rAAV6 capsid variants enable targeted gene delivery to microglia.
  • This represents the first successful instance of AAV-mediated gene expression and functional modulation in microglia.
  • These engineered vectors offer a promising platform for microglia-specific gene targeting in research and therapeutic applications for CNS diseases.