Spotlights on Recent JACS Publications

摘要

Hiroshi Sugiyama, Ganesh Pandian, and their co-workers report the development of organic ligands that localize in mitochondria and selectively bind to mitochondrial DNA, thereby advancing the field of mitochondrial gene therapy (DOI: 10.1021/ jacs.7b05230). The regulation of protein expression or activity using molecular ligands is the foundation of biochemical function across living organisms. Natural or synthetic ligands are also used extensively for the same purpose, as medical treatments for many diseases. However, mitochondrial diseases have been difficult to treat this way because of challenges with specificity and selectivity in delivery and site recognition. The authors have successfully combined a polyamide known to bind to DNA with a peptide sequence able to penetrate the mitochondrial membrane. By manipulating the polyamide sequence, the researchers can selectively control mitochondrial gene transcription in a dose-dependent manner.