Imaging of adeno-associated viral capsids for purposes of gene editing using CEST NMR/MRI.

ABSTRACT:

Purpose

Gene therapy using adeno-associated virus (AAV) vector-mediated gene delivery has undergone substantial growth in recent years with promising results in both preclinical and clinical studies, as well as emerging regulatory approval. However, the inability to quantify the efficacy of gene therapy from cellular delivery of gene-editing technology to specific functional outcomes is an obstacle for efficient development of gene therapy treatments. Building on prior works that used the CEST reporter gene lysine rich protein, we hypothesized that AAV viral capsids may generate endogenous CEST contrast from an abundance of surface lysine residues.

Methods

NMR experiments were performed on isolated solutions of AAV serotypes 1-9 on a Bruker 800-MHz vertical scanner. In vitro

SUBMITTER: Lam B 

PROVIDER: S-EPMC11142879 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature