Thermostability and in vivo performance of AAV9 in a film matrix.

ABSTRACT:

Background

Adeno-associated virus (AAV) vectors are stored and shipped frozen which poses logistic and economic barriers for global access to these therapeutics. To address this issue, we developed a method to stabilize AAV serotype 9 (AAV9) in a film matrix that can be stored at ambient temperature and administered by systemic injection.

Methods

AAV9 expressing the luciferase transgene was mixed with formulations, poured into molds and films dried under aseptic conditions. Films were packaged in individual particle-free bags with foil overlays and stored at various temperatures under controlled humidity. Recovery of AAV9 from films was determined by serial dilution of rehydrated film in media and infection of HeLa RC32 cells. Luciferase expression was compared to that of fi

SUBMITTER: Doan TNK 

PROVIDER: S-EPMC9681776 | biostudies-literature | 2022 Nov

REPOSITORIES: biostudies-literature