The present invention relates to a base
correction system that corrects mitochondrial
DNA mutations G3460A, G11778A, or T14484C, which are present in patients with Leber's hereditary
optic neuropathy (LHON), to a normal
genotype. Specifically, the present invention provides a base editor capable of correcting a
mutation site in a mitochondrial
gene of an LHON patient to a normal
genotype. The present invention also provides a method for correcting a mitochondrial
gene mutation using a
fusion protein or a
polynucleotide encoding such a
fusion protein that recognizes a specific site in the mitochondrial
gene of an LHON patient and specifically corrects the adenine base at position 3460, the adenine base at position 11778, or the
cytosine base at position 14484. The base editor or
polynucleotide according to the present invention can be used in cells or in an
extracellular test tube environment to correct
DNA mutations specifically expressed in LHON, and more preferably, can be used as a
gene therapy agent to prevent or treat the
disease. Thus, the present invention also provides a use of the substance for preventing or treating Leber's hereditary
optic neuropathy.