S Supanji, A B I Perdamaian, D K Paramita, R I Jenie
{"title":"基于临床和遗传学研究的一例罕见视网膜色素变性患者。","authors":"S Supanji, A B I Perdamaian, D K Paramita, R I Jenie","doi":"","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":39388,"journal":{"name":"Medical Journal of Malaysia","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2024-08-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"An uncommon case of retinitis pigmentosa patients based on clinical and genetic study.\",\"authors\":\"S Supanji, A B I Perdamaian, D K Paramita, R I Jenie\",\"doi\":\"\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":39388,\"journal\":{\"name\":\"Medical Journal of Malaysia\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-08-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Medical Journal of Malaysia\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Medical Journal of Malaysia","FirstCategoryId":"1085","ListUrlMain":"","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"Medicine","Score":null,"Total":0}
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.
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.