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The Progress of Gene Therapy for Leber's Optic Hereditary Neuropathy

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收录情况： ◇ SCIE

作者：

单位： [1]Hubei Univ Med, Taihe Hosp, Dept Ophthalmol Shiyan, Shiyan, Hubei, Peoples R China [2]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Ophthalmol,Wuhan,Hubei,Peoples R China

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关键词： Leber's optic hereditary neuropathy Gene therapy Translational medicine Mitochondrial disease MT-ND4 Allotopic expression technology

摘要：

Introduction: Leber's Optic Hereditary Neuropathy (LHON) is a common cause of teenaged blindness in both eyes for which there is currently no effective treatment. In 1871, the German ophthalmologist Theodor Leber was the first to describe the clinical characteristics of his namesake disease, and through unremitting efforts over the past 100 years, researchers have continued to increase their understanding of LHON. In recent years, using gene therapy, several groups have obtained breakthroughs in the treatment of the disease. Conclusion: In this article, we will review the challenging journey that researchers faced towards our current understanding of LHON, and describe the transition of gene therapy research for LHON from the bench to bedside.

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出版当年[2016]版：

大类 | 2 区医学

小类 | 3 区遗传学

最新[2025]版：

大类 | 4 区医学

小类 | 4 区遗传学

JCR分区：

出版当年[2015]版：

Q2 GENETICS & HEREDITY

最新[2023]版：

Q2 GENETICS & HEREDITY

影响因子： 3.8 最新[2023版] 3.2 最新五年平均 2.738 出版当年[2015版] 3.186 出版当年五年平均 2.542 出版前一年[2014版] 2.78 出版后一年[2016版]

第一作者：

第一作者单位： [1]Hubei Univ Med, Taihe Hosp, Dept Ophthalmol Shiyan, Shiyan, Hubei, Peoples R China

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通讯机构： [1]Hubei Univ Med, Taihe Hosp, Dept Ophthalmol Shiyan, Shiyan, Hubei, Peoples R China [2]Huazhong Univ Sci & Technol,Tongji Hosp,Tongji Med Coll,Dept Ophthalmol,Wuhan,Hubei,Peoples R China

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[1]Visual Field Variability after Gene Therapy for Leber's Hereditary Optic Neuropathy [2]Three Cases of Leber's Hereditary Optic Neuropathy with Rapid Increase in Visual Acuity After Gene Therapy [3]Evaluation of Leber's hereditary optic neuropathy patients prior to a gene therapy clinical trial [4]Long-term outcomes of gene therapy for the treatment of Leber's hereditary optic neuropathy [5]Seven-Year Follow-up of Gene Therapy for Leber's Hereditary Optic Neuropathy [6]Seven-Year Follow-up of Gene Therapy for Leber's Hereditary Optic Neuropathy [7]Clinical Observation of Patients with Leber's Hereditary Optic Neuropathy Before Gene Therapy [8]Factors associated with rapid improvement in visual acuity in patients with Leber's hereditary optic neuropathy after gene therapy [9]Prognostic factors for visual acuity in patients with Leber's hereditary optic neuropathy after rAAV2-ND4 gene therapy [10]Observation of Peripapillary Choroidal Vascularity in Natural Disease Course and After Gene Therapy for Leber's Hereditary Optic Neuropathy