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Related Experiment Video

Updated: Sep 22, 2025

Digital Droplet PCR Method for the Quantification of AAV Transduction Efficiency in Murine Retina
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Quantitative single-cell transcriptome-based ranking of engineered AAVs in human retinal explants.

Zhouhuan Xi1,2, Bilge E Öztürk1, Molly E Johnson1

  • 1Department of Ophthalmology, University of Pittsburgh, PA, USA.

Molecular Therapy. Methods & Clinical Development
|May 26, 2022
PubMed
Summary

This study used human retinal explants and single-cell RNA sequencing to evaluate adeno-associated virus (AAV) vectors for gene therapy. Top-performing AAVs were identified, accelerating the development of retinal gene therapies.

Keywords:
AAVgene therapyhuman retinal explantretinascRNA-seq

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Area of Science:

  • Ophthalmology
  • Molecular Biology
  • Gene Therapy

Background:

  • Adeno-associated viruses (AAVs) are key viral vectors for gene therapy, but predicting their clinical success is challenging due to cross-species differences.
  • Human retinal explants offer a unique model for studying gene delivery in fully developed human retinal tissue.

Purpose of the Study:

  • To evaluate the efficiency and tropism of 18 wild-type and engineered AAV capsids in human retinal explants.
  • To characterize the transcriptome of human retinal explants and assess AAV infectivity at single-cell resolution.

Main Methods:

  • Utilized a single-cell RNA sequencing (scAAVengr) pipeline to analyze AAV vector performance in human retinal explants.
  • Quantified AAV efficiency and tropism across different retinal cell types.
  • Validated top-performing AAV serotypes in non-human primate and human retinal explants.

Main Results:

  • Human retinal explants maintained major cell types with largely preserved gene expression, except for photoreceptors.
  • Single-cell resolution revealed differential infectivity and tropism of various AAV serotypes in human retinal cells.
  • AAV serotypes K91, K912, and 7m8 demonstrated superior performance in human retinal explants.

Conclusions:

  • Human retinal explants are a valuable model for AAV vector validation in retinal gene therapy.
  • This study provides critical data on AAV tropism in the human retina, aiding the translation of gene therapies.
  • Identified leading AAV serotypes that can accelerate the development of effective treatments for retinal diseases.