Section on Endocrinology and Genetics, Program on Developmental Endocrinology & Genetics (PDEGEN) & Pediatric Endocrinology Inter-Institute Training Program, Eunice Kennedy Shriver, National Institute of Child Health & Human Development (NICHD), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Section on Endocrinology and Genetics, Program on Developmental Endocrinology & Genetics (PDEGEN) & Pediatric Endocrinology Inter-Institute Training Program, Eunice Kennedy Shriver, National Institute of Child Health & Human Development (NICHD), National Institutes of Health (NIH), Bethesda, MD 20892, USA.
Mol Cell Endocrinol. 2014 Apr 5;386(1-2):85-91. doi: 10.1016/j.mce.2013.08.022. Epub 2013 Sep 5.
Endocrine neoplasia syndromes feature a wide spectrum of benign and malignant tumors of endocrine and non-endocrine organs associated with other clinical manifestations. This study outlines the main clinical features, genetic basis, and molecular mechanisms behind two multiple endocrine neoplasia syndromes that share quite a bit of similarities, but one can be inherited whereas the other is always sporadic, Carney complex (CNC) and McCune-Albright (MAS), respectively. Spotty skin pigmentation, cardiac and other myxomas, and different types of endocrine tumors and other characterize Carney complex, which is caused largely by inactivating Protein kinase A, regulatory subunit, type I, Alpha (PRKAR1A) gene mutations. The main features of McCune-Albright are fibrous dysplasia of bone (FD), café-au-lait macules and precocious puberty; the disease is caused by activating mutations in the Guanine Nucleotide-binding protein, Alpha-stimulating activity polypeptide (GNAS) gene which are always somatic. We review the clinical manifestations of the two syndromes and provide an update on their molecular genetics.
内分泌肿瘤综合征的特征是广泛的良性和恶性肿瘤的内分泌和非内分泌器官与其他临床表现。本研究概述了两个多发性内分泌肿瘤综合征的主要临床特征、遗传基础和分子机制,它们有很多相似之处,但一个是遗传性的,另一个是散发性的,分别是卡尼综合征(CNC)和麦卡恩-阿尔布赖特综合征(MAS)。斑驳的皮肤色素沉着、心脏和其他黏液瘤以及不同类型的内分泌肿瘤和其他特征是卡尼综合征,主要是由蛋白激酶 A 调节亚基,I 型,α(PRKAR1A)基因突变失活引起的。麦卡恩-阿尔布赖特综合征的主要特征是骨纤维发育不良(FD)、咖啡牛奶斑和性早熟;该病是由鸟嘌呤核苷酸结合蛋白,α刺激活性多肽(GNAS)基因的激活突变引起的,这些突变总是体细胞的。我们回顾了这两种综合征的临床表现,并对其分子遗传学进行了更新。