title: Novel therapies targeting inner mitochondrial membrane--from discovery to clinical development.
abstract: Mitochondrial oxidative stress and dysfunction have been implicated in the aging process and in numerous chronic diseases. The need for therapies that can protect and/or improve mitochondrial function is obvious. However, the development of mitoprotective drugs has been hampered by a number of challenges, and there are at present no approved therapies for mitochondrial dysfunction. This article describes the original discovery, preclinical development, and clinical development of a novel class of small peptide molecules that selectively target the inner mitochondrial membrane and protect mitochondrial function. These compounds have the potential to be a paradigm-shifting approach to the treatment of mitochondrial dysfunction, which underlies many common diseases, including cardiorenal, neurologic, and metabolic disorders.
authors.affiliation: Department of Pharmacology, Joan and Sanford I. Weill Medical College of Cornell University, 1300 York Avenue, New York, New York 10021, USA. hhszeto@med.cornell.edu
authors.forename: Hazel H
authors.initials: HH
authors.lastname: Szeto
authors.forename: Peter W
authors.initials: PW
authors.lastname: Schiller
mesh_terms: D055808:Drug Discovery; D004345:Drug Industry / Q000379:methods*; D006801:Humans; D008928:Mitochondria / Q000378:metabolism*; D058990:Molecular Targeted Therapy; D020011:Protective Agents / Q000378:metabolism / Q000494:pharmacology*
chemical_list: D020011:Protective Agents