Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Serial Pretreatment Evaluation from Baseline to Two Years

The preparatory phase of the LHON gene therapy trial aims to characterize affected patients and carriers for the planned gene therapy study that will utilize "allotopic expression," delivering copies of normal nuclear-encoded ND4 gene into the nuclei of retinal ganglion cells via an adeno-associated virus vector. The normal ND4 protein expressed in the cytoplasm is then imported into the mitochondria.