MITOCHONDRIAL OPTOGENETICS-BASED GENE THERAPY FOR TREATING CANCER

摘要

Disclosed herein is an optogenetics-based gene therapy that involves channelrhodopsin fusion proteins having an inner mitochondrial membrane-mitochondrial localization signal (IMM-MLS) that can effectively target the fusion protein to an inner mitochondria membrane, and a channelrhodopsin ion channel domain that can change the mitochondrial membrane potential (ΔΨm) when light is present. The disclosed optogenetics-based gene therapy system can in some embodiments further involve luciferase fusion proteins to stimulate the channelrhodopsin without reliance on external light that has an outer mitochondrial membrane-mitochondrial localization signal (OMM-MLS) that can effectively target the luciferase fusion protein to an outer mitochondrial membrane, and a luciferase protein that can produce a bioluminescence in the presence of a luciferase substrate.