QUANTUM DOT ENCAPSULATED VIRAL LIKE PARTICLES BASED ON ADENO- ASSOCIATED VIRUS

Abstract

Adeno-associated virus (AAV) has emerged as a leading gene-therapy candidate due to its broad tissue tropism and encouraging safety profile. Traditionally, viral biodistribution is assessed through expression of reporter genes in tissues days to weeks post-vector administration. This does not provide insight into how AAV initially distributes throughout the body which will impact its effectiveness and long-term safety. Towards this end, we optimized a strategy for assembling a viral like particle (VLP) using capsid proteins from AAV serotype 2 and incorporated a quantum dot (QD) into its core. Their uptake was visualized in human embryonic kidney (HEK) cells in vitro. Our findings enable further optimization of VLP-QD systems for AAV biodistribution analysis. In addition, extracellular vesicles (EVs) are a new class of gene-delivery vector with potential in pulmonary disease. We measured the diffusion of EVs from two cell lines in human mucus to determine their potential as an inhaled therapeutic.