Happle R
J Am Acad Dermatol. 1987 Apr;16(4):899-906. doi: 10.1016/s0190-9622(87)80249-9.
A genetic concept is advanced to explain the origin of several sporadic syndromes characterized by a mosaic distribution of skin defects. It is postulated that these disorders are due to the action of a lethal gene surviving by mosaicism. The presence of the mutation in the zygote will lead to death of the embryo at an early stage of development. Cells bearing the mutation can survive only in a mosaic state, in close proximity with normal cells. The mosaic may arise either from a gametic half chromatid mutation or from an early somatic mutation. This concept of origin is proposed to apply to the Schimmelpenning-Feuerstein-Mims syndrome, the McCune-Albright syndrome, the Klippel-Trenaunay syndrome, the Sturge-Weber syndrome, and neurocutaneous melanosis. Moreover, this etiologic hypothesis may apply to two other birth defects that have recently been delineated, the Proteus syndrome (partial gigantism of hands or feet, hemihypertrophy, macrocephaly, linear papillomatous epidermal nevus, subcutaneous hemangiomas and lipomas, accelerated growth, and visceral anomalies), and the Delleman-Oorthuys syndrome (orbital cyst, porencephaly, periorbital appendages, and focal aplasia of the skin.
提出了一种遗传学概念来解释几种以皮肤缺陷呈镶嵌分布为特征的散发性综合征的起源。据推测,这些疾病是由于一个通过镶嵌现象存活的致死基因的作用。合子中突变的存在会导致胚胎在发育早期死亡。携带突变的细胞只能以镶嵌状态存活,与正常细胞紧密相邻。这种镶嵌现象可能源于配子半染色单体突变或早期体细胞突变。提出的这种起源概念适用于施密尔彭宁 - 费尔斯坦 - 米姆斯综合征、麦库恩 - 奥尔布赖特综合征、克-特综合征、斯-韦综合征和神经皮肤黑素沉着症。此外,这种病因假说可能适用于最近已被描述的另外两种出生缺陷,即变形综合征(手足部分巨大症、偏侧肥大、巨头畸形、线状乳头状表皮痣、皮下血管瘤和脂肪瘤、生长加速以及内脏异常)和德-奥综合征(眼眶囊肿、脑穿通畸形、眶周附属器以及皮肤局灶性发育不全)。