Department of Pediatrics, Division of Genetics and Genomic Medicine, Washington University School of Medicine, St. Louis, Missouri.
Department of Pediatrics, Division of Endocrinology and Diabetes and the Division of Bone and Mineral Diseases; Washington University School of Medicine, St. Louis, Missouri.
Mo Med. 2024 Jul-Aug;121(4):297-303.
Metabolic bone diseases are a heterogenous group of conditions that all result in aberrant bone mineral homeostasis with resulting skeletal disease. The underlying causes are variable, ranging from nutritional deficiencies to pathogenic variants in skeletal genes. To properly diagnose and treat these conditions, a clinician needs to understand bone metabolism as well as recognize the signs of disease in a patient. This review will focus on three relatively common metabolic bone diseases (osteogenesis imperfecta, hypophosphatasia, and X-linked hypophosphatemic rickets) that are caused by genetic variants, not by nutritional deficiency. As molecular DNA sequencing has improved, the scientific community has been able to better understand the genetic basis of these conditions and create sophisticated medical treatments based on the genetic deficiency.
代谢性骨病是一组异质性疾病,它们都会导致骨矿物质稳态异常,从而引发骨骼疾病。其根本原因多种多样,从营养缺乏到骨骼基因的致病变异都有。为了正确诊断和治疗这些疾病,临床医生不仅需要了解骨代谢,还需要识别患者的疾病迹象。这篇综述将重点介绍三种相对常见的代谢性骨病(成骨不全症、低磷酸酶血症和 X 连锁低磷血症性佝偻病),这些疾病是由遗传变异引起的,而不是由营养缺乏引起的。随着分子 DNA 测序技术的改进,科学界已经能够更好地理解这些疾病的遗传基础,并根据遗传缺陷制定出复杂的医疗方法。