基于临床和遗传学研究的一例罕见视网膜色素变性患者。

Q3 Medicine Medical Journal of Malaysia Pub Date : 2024-08-01
S Supanji, A B I Perdamaian, D K Paramita, R I Jenie
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引用次数: 0

摘要

遗传性视网膜营养不良症(IRD)是由视觉通路相关基因突变引起的一组表型,主要发生在光感受器上。这组不同类型的病症包括以视网膜周边骨刺为特征的色素性视网膜炎(RP),以及以黄斑鱼尾状斑点为特征的斯塔加特病(Stargardt)。在这项研究中,一名 20 岁的男性患者伴有视网膜色素变性的症状,黄斑上有淡黄色梭形皱襞。然而,他的兄弟、母亲和姨妈却患有典型的斯塔加特病。这项研究涉及四人,两名男性(病例 1 和 2)、他们的母亲(病例 3)和姨妈(病例 4)。病例 1 和病例 2 初诊时,病例 1 被诊断为 RP 和黄斑营养不良,病例 2 被诊断为斯塔加特病。随访期间,病例 1 和 2 及其父亲、母亲和其他家庭成员接受了全面的眼部检查,包括眼底、Snellen、OCT、OCT-A 和 HFA。病例 1 的父亲是健康的,而母亲和他的一位姨妈被诊断为斯塔加特眼病。对病例 1 进行了遗传学分析,发现与 IRD 相关的多种基因突变,其中锥体蛋白编码基因突变集中在视网膜中央,杆状蛋白编码基因突变集中在视网膜周边。这种 RP 表型是否由多个或相同突变组合造成,还需要进一步分析和验证。病例 2 和病例 3 的基因分析显示了类似的突变结果,但其周边视网膜是健康的,仅代表 Stargardt。病例 1 被认为是伴有黄斑营养不良的 RP,而病例 2、3 和 4 被确认为 Stargardt。
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An uncommon case of retinitis pigmentosa patients based on clinical and genetic study.

Inherited retinal dystrophy (IRD) is a group of phenotypes caused by mutations in visual pathways-related genes, mostly occurring at photoreceptors. This heterogeneous group includes retinitis pigmentosa (RP) recognised by bone spicule at the peripheral retina and the other is Stargardt with macular pisiform flecks. In this study, a 20- year-old male patient with RP symptoms was accompanied by a yellowish pisiform flex in the macula. However, his brother, mother and aunty have typical Stargardt disease. This study involved four persons, two males (cases 1 and 2), their mother (case 3) and aunt (case 4). Initially, cases 1 and 2 came to the clinic, case 1 was diagnosed as RP and macular dystrophy, and case 2 was diagnosed as Stargardt disease. On the follow-up, cases 1 and 2 as well as their father, mother and other family members underwent comprehensive eye examination, including fundus, Snellen, OCT, OCT-A and HFA, and found an uncommon macular abnormality besides typical RP appearance in case 1. The father is healthy while the mother and one of his aunties were diagnosed as Stargardt. A genetics analysis was conducted in case 1, finding various mutations associated with IRD mutation at the cone protein-encoded gene that concentrated at the central and rod protein-encoded gene concentrated at the peripheral retina. Whether the combination of multiple or the same mutations is responsible for this RP phenotype needs further analysis and validation. Cases 2 and 3 genetic analysis showed similar mutation results but with a healthy peripheral retina and only represented Stargardt. Case 1 is considered as RP with macular dystrophy, while cases 2, 3 and 4 are confirmed as Stargardt.

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来源期刊
Medical Journal of Malaysia
Medical Journal of Malaysia Medicine-Medicine (all)
CiteScore
1.20
自引率
0.00%
发文量
165
期刊介绍: Published since 1890 this journal originated as the Journal of the Straits Medical Association. With the formation of the Malaysian Medical Association (MMA), the Journal became the official organ, supervised by an editorial board. Some of the early Hon. Editors were Mr. H.M. McGladdery (1960 - 1964), Dr. A.A. Sandosham (1965 - 1977), Prof. Paul C.Y. Chen (1977 - 1987). It is a scientific journal, published quarterly and can be found in medical libraries in many parts of the world. The Journal also enjoys the status of being listed in the Index Medicus, the internationally accepted reference index of medical journals. The editorial columns often reflect the Association''s views and attitudes towards medical problems in the country. The MJM aims to be a peer reviewed scientific journal of the highest quality. We want to ensure that whatever data is published is true and any opinion expressed important to medical science. We believe being Malaysian is our unique niche; our priority will be for scientific knowledge about diseases found in Malaysia and for the practice of medicine in Malaysia. The MJM will archive knowledge about the changing pattern of human diseases and our endeavours to overcome them. It will also document how medicine develops as a profession in the nation. We will communicate and co-operate with other scientific journals in Malaysia. We seek articles that are of educational value to doctors. We will consider all unsolicited articles submitted to the journal and will commission distinguished Malaysians to write relevant review articles. We want to help doctors make better decisions and be good at judging the value of scientific data. We want to help doctors write better, to be articulate and precise.
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