Intracellular Delivery of Recombinant Human Proteins into Mammalian cells as a Potential Therapeutic Approach

摘要

A large number of genetic diseases are characterized by missing or malfunctioned intracellular proteins encoded by mutated genes. A potential therapeutic approach for such disorders would be targeted intracellular delivery of physiological form of the missing protein, since proteins are not permeable through biological membranes. The discovery that protein transduction domains (PTDs) are able to facilitate intracellular transduction of recombinant proteins has revolutionized this field. We wish to present the biotechnological production and subsequent delivery of recombinant mitochondrial proteins carrying PTDs into the mitochondria of mammalian cells.