Emerging therapeutic approaches to mitochondrial diseases.
Mitochondrial diseases are very heterogeneous and can affect different tissues and organs. Moreover, they can be caused by genetic defects in either nuclear or mitochondrial DNA as well as by environmental factors. All of these factors have made the development of therapies difficult. In this review article, we will discuss emerging approaches to the therapy of mitochondrial disorders, some of which are targeted to specific conditions whereas others may be applicable to a more diverse group of patients.
Wenz
Tina
T
Department of Neurology, University of Miami School of Medicine, 1095 NW 14th Terrace, Miami, FL 33136, USA.
Williams
Sion L
SL
Bacman
Sandra R
SR
Moraes
Carlos T
CT
D000328:Adult; D004252:DNA Mutational Analysis; D004272:DNA, Mitochondrial / Q000235:genetics; D002658:Developmental Disabilities / Q000235:genetics / Q000628:therapy*; D006580:Genetic Carrier Screening; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D028361:Mitochondrial Diseases / Q000235:genetics / Q000628:therapy*; D010085:Oxidative Phosphorylation
D004272:DNA, Mitochondrial