Leber hereditary optic neuropathy (LHON) is one of the most common mitochondrial diseases mainly caused by mitochondrial DNA mutations and which usually cause rapid and painless onset of loss of bilateral vision. Effective treatments and preventions are not available. More than 150 years after LHON was first reported, and more than 30 years after discovering LHON associated with mtDNA mutations, we still not make a major breakthrough on the research, mainly hampered by the lack of appropriate models. This paper mainly focuses on the latest research results of LHON and the new progress of disease prevention and treatment, aiming to further strengthen the understanding of this disease.