Conventional and Tropism-Modified High-Capacity Adenoviral Vectors Exhibit Similar Transduction Profiles in Human iPSC-Derived Retinal Organoids

Research Overview

Viral vectors are a promising route for retinal gene therapy, but adeno-associated vectors — the primary platform — carry too little cargo for several clinically relevant retinal genes. This study tested high-capacity adenoviral vectors, which hold up to 36 kb and could accommodate every known retinal gene coding sequence.

Vectors built on classical adenovirus type 5 and on a fiber-modified AdV5.F50 variant each delivered a 29.6 kb genome encoding a fluorescent reporter. Their tropism was evaluated in iPSC-derived human retinal organoids, and both vector types demonstrated transduction of the organoid tissue, establishing the platform’s feasibility for large retinal genes.