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Clinical Observation of Patients with Leber's Hereditary Optic Neuropathy before Gene Therapy.

Current Gene Therapy 2018 November 6
BACKGROUND: Leber's hereditary optic neuropathy is a hereditary disease inherited from mitochondria. So far, there is no effective treatment, and gene therapy has become the most promising and promising method. Because of the possibility of spontaneous visual acuity recovery in this disease, we screened the patients in before gene therapy, excluding those with spontaneous visual acuity improvement, and prepared for the subsequent gene therapy.

OBJECTIVE: To clinically observe the course of Leber's hereditary optic neuropathy for 6 months prior to gene therapy.

METHOD: Sixty-six patients with Leber's hereditary optic neuropathy were enrolled in the study. Patients were classified based on duration of disease: less than 24 months and over 24 months. Three clinical follow-up examinations were conducted over 1 year. We assessed intraocular pressure, visual acuity, visual field, retinal nerve fiber layer thickness, fundus photographs, and visual evoked potential.

RESULTS: Eighty-two eyes of 34 patients displayed stable visual acuity; 33 eyes of 22 patients displayed decrease in visual acuity (less than 24 months: 24 eyes; over 24 months: nine eyes); and 17 eyes of 12 patients showed improvement in visual acuity (less than 24 months: four eyes; over 24 months: 13 eyes). Visual acuity and visual field indices decreased over 24 months from disease onset and appeared stable after 24 months.

CONCLUSION: Most patients of Leber's hereditary optic neuropathy gradually stabilize visual function with prolonged onset time, and the lower possibility of spontaneous vision recovery provides a basis for future evaluation of the effectiveness of gene therapy.

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