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Related Experiment Video

Updated: Apr 27, 2026

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

Retinal transduction profiles by high-capacity viral vectors.

A Puppo¹, G Cesi¹, E Marrocco¹

¹Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy.

|July 4, 2014

Summary

Adeno-associated viral vectors are limited for inherited retinopathies due to large gene sizes. New viral vectors show potential for retinal gene therapy, but photoreceptor transduction remains a challenge.

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Area of Science:

Ophthalmology
Gene Therapy
Molecular Biology

Background:

Adeno-associated viral (AAV) vectors are established for retinal gene therapy.
AAV's limited cargo capacity restricts treatment for inherited retinopathies (IRs) caused by large genes (>5kb).
Alternative viral vectors like adenovirus (Ad), lentivirus (LV), and herpes virus (HV) can carry larger payloads but show inefficient retinal photoreceptor (PR) targeting.

Purpose of the Study:

To evaluate and compare the retinal transduction efficiency of various viral vectors beyond AAV.
To identify Ad, LV, and HV vectors capable of targeting retinal photoreceptors for IR gene therapy.
To assess the potential of these vectors for treating IRs linked to large gene mutations.

Main Methods:

Systematic evaluation of mouse retinal transduction profiles.
Testing of 16 Ad serotypes, 7 LV pseudotypes, and a bovine HV.
Comparative analysis of photoreceptor (PR) and retinal pigment epithelium (RPE) transduction.

Main Results:

Most tested vectors efficiently transduced the retinal pigment epithelium (RPE).
Lentivirus-GP64 showed improved PR transduction compared to LV-VSVG, but in a limited region.
Certain adenovirus vectors (HdAd1, 2, 5/F35++) demonstrated more extensive PR transduction than LV, though not surpassing AAV2/8.

Conclusions:

Adenovirus vectors show promise for enhanced photoreceptor transduction in retinal gene therapy.
Further optimization is needed to match AAV's broad PR transduction for treating inherited retinopathies.
Exploring novel viral vector systems is crucial for overcoming AAV cargo limitations in gene therapy.