The Progress of Gene Therapy for Lebers Optic Hereditary Neuropathy

摘要

Introduction: Leber’s Optic Hereditary Neuropathy (LHON) is a common cause of teenaged blindness in both eyes for which there is currently no effective treatment. In 1871, the German ophthal-mologist Theodor Leber was the first to describe the clinical characteristics of his namesake disease, and through unremitting efforts over the past 100 years, researchers have continued to increase their under-standing of LHON. In recent years, using gene therapy, several groups have obtained breakthroughs in the treatment of the disease.