Yuan Jia-Jia, Zhang Yong, Wang Li-Ling, Cheng Ming-Shi, Ma Si-Qi, Gao Qin, Li Bin
Department of Ophthalmology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Department of Ophthalmology, Taihe Hospital, Hubei University of Medicine, Shiyan, China.
Ophthalmic Res. 2018;60(3):176-184. doi: 10.1159/000487485. Epub 2018 Apr 12.
To assess changes in visual field (VF) values after gene therapy for Leber's hereditary optic neuropathy (LHON).
VF recovery, VF indices, and mean deviation in injected and uninjected eyes, before and after gene therapy, were examined in 2 groups of patients according to disease duration (≤2 years and > 2 years). Nine patients with LHON were treated by monocular intravitreal injection of AAV2-ND4. Finally, 7 patients were considered for subsequent comparisons; the first and second eyes were treated separately.
There were no significant differences in VF indices and mean deviation between injected and uninjected eyes (p = 0.910 and p = 0.929, respectively). However, there was a significant difference before and after injection (p = 0.016 and p = 0.015, respectively). There was no significant difference in VF improvement between patients with ≤2 years' disease duration and those with a longer disease duration.
There was a statistically significant VF improvement after gene therapy. This suggests that monocular intravitreal injection of AAV2-ND4 can improve binocular VF values. This study also suggests that gene therapy can be effective in patients with a disease duration of > 2 years.
评估基因治疗对Leber遗传性视神经病变（LHON）患者视野（VF）值的影响。
根据病程（≤2年和>2年）将两组患者分为两组，检测基因治疗前后注射眼和未注射眼的VF恢复情况、VF指数和平均偏差。9例LHON患者接受了单眼玻璃体内注射AAV2-ND4治疗。最后，7例患者被纳入后续比较；先后分别对第一只眼和第二只眼进行治疗。
注射眼和未注射眼的VF指数和平均偏差无显著差异（p分别为0.910和0.929）。然而，注射前后存在显著差异（p分别为0.016和0.015）。病程≤2年的患者与病程较长的患者在VF改善方面无显著差异。
基因治疗后VF有统计学意义的改善。这表明单眼玻璃体内注射AAV2-ND4可改善双眼VF值。本研究还表明，基因治疗对病程>2年的患者可能有效。
