The Co-Transduction Conundrum: Improving Dual rAAV Delivery Through Co-Administration of Capsid Serotypes With Complementary Tropism.

ABSTRACT:

Purpose

Recombinant adeno-associated virus (rAAV) vectors are promising tools for the treatment of inherited retinal diseases (IRDs) but have a limited carrying capacity for therapeutic cassettes of up to 4.8 kilobase (kb). To circumvent this limitation, multiple vector approaches have been proposed wherein the transgene cassette is split across two or more rAAV vector genomes. In this study, we examine whether rAAV serotype choice has an effect on co-transduction of retinal cells following subretinal delivery and whether co-transduction represents a rate-limiting step preventing successful large transgene delivery.

Methods

The rAAV vector serotypes with known photoreceptor affinity (rAAV2/5, rAAV2/8, and rAAV2/9) packaging fluorescent reporter genes (GFP or mCherry) were co

SUBMITTER: Fehrman RL 

PROVIDER: S-EPMC12663885 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature