Advancements in LHON Gene Therapy

This chapter explores the complexities of gene therapy for Leber Hereditary Optic Neuropathy (LHON), emphasizing methodologies like viral vectors and specific mutation targets such as the 11778 mutation. It covers the structure and findings of phase three clinical trials, including the significant benefits of bilateral injections and the role of community support in patient recruitment. Additionally, the chapter discusses future clinical trials and the importance of educating patients to prevent misinformation about LHON.