Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

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

Frataxin deficiency drives cardiac dysfunction and transcriptional dysregulation in Friedreich ataxia iPSC model.

Cell death & disease·2026

Same author

Functional maturation of enteric neurons derived from human induced pluripotent stem cells.

Stem cell reports·2026

Same author

Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia models.

Cell reports. Medicine·2026

Same author

Patient induced pluripotent stem cells identify specificities of a reticular pseudodrusen phenotype in age-related macular degeneration.

Genome medicine·2026

Same author

Single-cell analysis reveals cellular heterogeneity and limits of marker-based assessment in retinal ganglion cell-enriched organoid cultures.

Molecular medicine (Cambridge, Mass.)·2026

Same author

Long-read sequencing-based atlas of tissue-specific expression of DNM1L transcript variants.

The FEBS journal·2026

Search research articles

Related Experiment Video

Updated: Jul 8, 2025

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease

Published on: April 19, 2015

AAV capsid bioengineering in primary human retina models.

Adrian Westhaus^1,2,3, Steven S Eamegdool⁴, Milan Fernando⁵

¹Translational Vectorology Research Unit, Faculty of Medicine and Health, Children's Medical Research Institute, The University of Sydney, Westmead, Australia.

Scientific Reports

|December 11, 2023

Summary

This study developed new adeno-associated viral (AAV) vectors for retinal gene therapy using human retinal explants. These novel AAV variants show high transgene expression, advancing preclinical models for gene therapy development.

More Related Videos

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine Retina

Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine Retina

Published on: December 25, 2021

Related Experiment Videos

Last Updated: Jul 8, 2025

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease

Using Adeno-associated Virus as a Tool to Study Retinal Barriers in Disease

Published on: April 19, 2015

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine Retina

Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine Retina

Published on: December 25, 2021

Area of Science:

Ophthalmology
Molecular Biology
Biotechnology

Background:

Adeno-associated viral (AAV) vector-mediated gene therapy is a promising approach for retinal diseases.
Developing novel AAV variants with improved properties is crucial for advancing clinical applications.
Predictive preclinical models are essential for validating new AAV vectors before human trials.

Purpose of the Study:

To evaluate the utility of primary retinal explant cultures for AAV capsid development.
To screen existing AAV capsids and develop novel variants for enhanced transduction of human retinal cells.
To compare the efficacy of newly developed AAV variants against established benchmarks in various retinal models.

Main Methods:

High-throughput screening of 51 AAV capsids in primary human retinal explants and other human retinal models.
Application of transgene expression-based directed evolution to engineer novel AAV capsids.
Side-by-side comparison of novel and benchmark AAV variants in four in vitro and ex vivo human retinal models.

Main Results:

Primary retinal explant cultures were found to be effective for AAV capsid screening and development.
Novel AAV variants were successfully engineered for more efficient transduction of primary human retinal cells.
The newly developed AAV variants demonstrated high transgene expression in primary human retinal cells, outperforming existing benchmarks in specific models.

Conclusions:

Primary retinal explant culture is a valuable tool for AAV capsid development in retinal gene therapy.
Novel AAV variants with superior transduction efficiency in human retinal cells have been identified.
These findings support the advancement of AAV gene therapy for inherited retinal diseases.