Savage Sharon A, Bertuch Alison A
Clinical Genetics Branch, Division of Cancer Epidemiology and Genetics, National Cancer Institute, Bethesda, Maryland, USA.
Baylor College of Medicine, Houston, Texas, USA.
Br J Haematol. 2025 Jan;206(1):69-73. doi: 10.1111/bjh.19851. Epub 2024 Oct 27.
Rare germline pathogenic variants (GPVs) in genes essential in telomere length maintenance and function have been implicated in two broad classes of human disease. The telomere biology disorders (TBDs) are a spectrum of life-threatening conditions, including bone marrow failure, liver and lung disease, cancer and other complications caused by GPVs in telomere maintenance genes that result in short and/or dysfunctional telomeres and reduced cellular replicative capacity. In contrast, cancer predisposition with long telomeres (CPLT) is a disorder associated with elevated risk of a variety of cancers, primarily melanoma, thyroid cancer, sarcoma, glioma and lymphoproliferative neoplasms caused by GPVs in shelterin complex genes that lead to excessive telomere elongation and increased cellular replicative capacity. While telomeres are at the root of both disorders, the term TBD is used to convey the clinical phenotypes driven by critically short or otherwise dysfunctional telomeres and their biological consequences.
端粒长度维持和功能所必需的基因中的罕见种系致病变体(GPV)与两大类人类疾病有关。端粒生物学障碍(TBD)是一系列危及生命的疾病,包括骨髓衰竭、肝脏和肺部疾病、癌症以及由端粒维持基因中的GPV导致的其他并发症,这些基因会导致端粒短缩和/或功能异常,并降低细胞复制能力。相比之下,长端粒癌症易感性(CPLT)是一种与多种癌症风险升高相关的疾病,主要是黑色素瘤、甲状腺癌、肉瘤、神经胶质瘤和淋巴增殖性肿瘤,由保护素复合体基因中的GPV引起,导致端粒过度延长和细胞复制能力增加。虽然端粒是这两种疾病的根源,但术语TBD用于描述由严重短缩或功能异常的端粒及其生物学后果所驱动的临床表型。