{"title":"产前诊断hspg2相关的节段性发育不良:来自土耳其的第一份报告。","authors":"Mehmet Berkay Akcan, Raziye Torun, Tuba Sözen Türk, Özgür Kırbıyık, Atalay Ekin, Altuğ Koç","doi":"10.1002/ajmg.a.64152","DOIUrl":null,"url":null,"abstract":"<p><p>Silverman-Handmaker type dyssegmental dysplasia (DDSH) is a rare and lethal skeletal dysplasia caused by biallelic null variations in the HSPG2 gene, which encodes the extracellular matrix proteoglycan perlecan. Here, we report a prenatal case of DDSH identified at 18 weeks of gestation, referred due to ultrasonographic findings of limb shortening, retrognathia, and irregularities in the lumbar vertebrae. Targeted skeletal dysplasia panel testing via next-generation sequencing (NGS) revealed a novel homozygous splice site variant, c.1355 + 1 G>T, located at the canonical donor site of intron 11 in HSPG2. The fetus died shortly after birth, consistent with the expected DDSH phenotype. Our case expands the mutational spectrum of HSPG2-related skeletal dysplasias and underscores the diagnostic and prognostic challenges of novel splicing variants in prenatal genetic counseling. It also emphasizes the value of combining prenatal imaging with molecular diagnostics to improve diagnostic accuracy and support informed reproductive decision-making.</p>","PeriodicalId":7507,"journal":{"name":"American Journal of Medical Genetics Part A","volume":" ","pages":"e64152"},"PeriodicalIF":1.7000,"publicationDate":"2025-06-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Prenatal Diagnosis of HSPG2-Related Dyssegmental Dysplasia: The First Report From Turkey.\",\"authors\":\"Mehmet Berkay Akcan, Raziye Torun, Tuba Sözen Türk, Özgür Kırbıyık, Atalay Ekin, Altuğ Koç\",\"doi\":\"10.1002/ajmg.a.64152\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Silverman-Handmaker type dyssegmental dysplasia (DDSH) is a rare and lethal skeletal dysplasia caused by biallelic null variations in the HSPG2 gene, which encodes the extracellular matrix proteoglycan perlecan. Here, we report a prenatal case of DDSH identified at 18 weeks of gestation, referred due to ultrasonographic findings of limb shortening, retrognathia, and irregularities in the lumbar vertebrae. Targeted skeletal dysplasia panel testing via next-generation sequencing (NGS) revealed a novel homozygous splice site variant, c.1355 + 1 G>T, located at the canonical donor site of intron 11 in HSPG2. The fetus died shortly after birth, consistent with the expected DDSH phenotype. Our case expands the mutational spectrum of HSPG2-related skeletal dysplasias and underscores the diagnostic and prognostic challenges of novel splicing variants in prenatal genetic counseling. It also emphasizes the value of combining prenatal imaging with molecular diagnostics to improve diagnostic accuracy and support informed reproductive decision-making.</p>\",\"PeriodicalId\":7507,\"journal\":{\"name\":\"American Journal of Medical Genetics Part A\",\"volume\":\" \",\"pages\":\"e64152\"},\"PeriodicalIF\":1.7000,\"publicationDate\":\"2025-06-12\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"American Journal of Medical Genetics Part A\",\"FirstCategoryId\":\"99\",\"ListUrlMain\":\"https://doi.org/10.1002/ajmg.a.64152\",\"RegionNum\":4,\"RegionCategory\":\"生物学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"GENETICS & HEREDITY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"American Journal of Medical Genetics Part A","FirstCategoryId":"99","ListUrlMain":"https://doi.org/10.1002/ajmg.a.64152","RegionNum":4,"RegionCategory":"生物学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}
Prenatal Diagnosis of HSPG2-Related Dyssegmental Dysplasia: The First Report From Turkey.
Silverman-Handmaker type dyssegmental dysplasia (DDSH) is a rare and lethal skeletal dysplasia caused by biallelic null variations in the HSPG2 gene, which encodes the extracellular matrix proteoglycan perlecan. Here, we report a prenatal case of DDSH identified at 18 weeks of gestation, referred due to ultrasonographic findings of limb shortening, retrognathia, and irregularities in the lumbar vertebrae. Targeted skeletal dysplasia panel testing via next-generation sequencing (NGS) revealed a novel homozygous splice site variant, c.1355 + 1 G>T, located at the canonical donor site of intron 11 in HSPG2. The fetus died shortly after birth, consistent with the expected DDSH phenotype. Our case expands the mutational spectrum of HSPG2-related skeletal dysplasias and underscores the diagnostic and prognostic challenges of novel splicing variants in prenatal genetic counseling. It also emphasizes the value of combining prenatal imaging with molecular diagnostics to improve diagnostic accuracy and support informed reproductive decision-making.
期刊介绍:
The American Journal of Medical Genetics - Part A (AJMG) gives you continuous coverage of all biological and medical aspects of genetic disorders and birth defects, as well as in-depth documentation of phenotype analysis within the current context of genotype/phenotype correlations. In addition to Part A , AJMG also publishes two other parts:
Part B: Neuropsychiatric Genetics , covering experimental and clinical investigations of the genetic mechanisms underlying neurologic and psychiatric disorders.
Part C: Seminars in Medical Genetics , guest-edited collections of thematic reviews of topical interest to the readership of AJMG .