Efficient spatially targeted gene editing using a near-infrared activatable protein-conjugated nanoparticle for brain applications.

ABSTRACT: Spatial control of gene expression is critical to modulate cellular functions and deconstruct the function of individual genes in biological processes. Light-responsive gene-editing formulations have been recently developed; however, they have shown limited applicability in vivo due to poor tissue penetration, limited cellular transfection and the difficulty in evaluating the activity of the edited cells. Here, we report a formulation composed of upconversion nanoparticles conjugated with Cre recombinase enzyme through a photocleavable linker, and a lysosomotropic agent that facilitates endolysosomal escape. This formulation allows in vitro spatial control in gene editing after activation with near-infrared light. We further demonstrate the potential of this formulation in vivo through thr

SUBMITTER: Rebelo C 

PROVIDER: S-EPMC9287341 | biostudies-literature | 2022 Jul

REPOSITORIES: biostudies-literature