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SSBP1 相关性视神经萎缩与黄斑病变的特征。

Characterization of SSBP1-related optic atrophy and foveopathy.

机构信息

National reference centre for inherited sensory diseases, University Hospital of Montpellier, University of Montpellier, Montpellier, France.

Sensgene Care Network, Strasbourg, France.

出版信息

Sci Rep. 2021 Sep 21;11(1):18703. doi: 10.1038/s41598-021-98150-1.

Abstract

Dominant optic atrophy (DOA) is genetically heterogeneous and most commonly caused by mutations in OPA1. To distinguish between the classical OPA1-related and the recently identified SSBP1-related DOAs, the retina and fovea of 27 patients carrying the SSBP1 p.Arg38Gln variant were scrutinized using 20° × 20° macular cube and 30° and 55° field fundus autofluorescence photographs. Age of onset, visual acuity, retinal nerve fiber layer and macular thicknesses were recorded. Three SSBP1-patients were asymptomatic, 10 had isolated DOA, and 12 had a combined DOA plus foveopathy. The foveopathy, with a tiny defect of the ellipsoid and interdigitation lines, was similar in all patients, independent of age. There were no significant statistical differences in terms of visual acuity and SD-OCT measurements between patients with isolated DOA (mean visual acuity in decimals: 0.54 ± 0.41) and those with combined foveopathy (0.50 ± 0.23). Two patients over 50 years of age developed a progressive rod-cone dystrophy, leading to severe visual impairment. SSBP1-related DOA shares similarities with OPA1-related DOA with an incomplete penetrance and an early childhood visual impairment. Nevertheless, the presence of a congenital foveopathy with no impact on visual acuity is a major criterion to distinguish SSBP1 cases and orient the appropriate genetic analysis.

摘要

显性视神经萎缩(DOA)具有遗传异质性,最常见的原因是 OPA1 突变。为了区分经典的 OPA1 相关 DOA 和最近发现的 SSBP1 相关 DOA,对携带 SSBP1 p.Arg38Gln 变异的 27 名患者的视网膜和黄斑进行了 20°×20° 黄斑立方和 30° 和 55° 视野眼底自发荧光照片检查。记录发病年龄、视力、视网膜神经纤维层和黄斑厚度。3 名 SSBP1 患者无症状,10 名孤立性 DOA,12 名合并 DOA 加黄斑病变。所有患者的黄斑病变均具有小的椭圆体和交织线缺陷,与年龄无关。孤立性 DOA 患者(十进制平均视力:0.54±0.41)和合并性黄斑病变患者(0.50±0.23)之间的视力和 SD-OCT 测量值没有显著统计学差异。两名 50 岁以上的患者出现进行性视杆-视锥细胞营养不良,导致严重视力损害。SSBP1 相关 DOA 与 OPA1 相关 DOA 具有相似性,表现为不完全外显率和儿童早期视力损害。然而,存在先天性黄斑病变而不影响视力是区分 SSBP1 病例并指导适当的基因分析的主要标准。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/fb2c/8455542/b5549e2e65fa/41598_2021_98150_Fig1_HTML.jpg

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