Department of Medicine (Cardiology), Duke University Medical Center, Durham, North Carolina, USA.
J Clin Invest. 2013 Apr;123(4):1638-46. doi: 10.1172/JCI66903. Epub 2013 Mar 15.
The identification of a gain-of-function mutation in CACNA1C as the cause of Timothy Syndrome (TS), a rare disorder characterized by cardiac arrhythmias and syndactyly, highlighted unexpected roles for the L-type voltage-gated Ca2+ channel CaV1.2 in nonexcitable cells. How abnormal Ca2+ influx through CaV1.2 underlies phenotypes such as the accompanying syndactyly or craniofacial abnormalities in the majority of affected individuals is not readily explained by established CaV1.2 roles. Here, we show that CaV1.2 is expressed in the first and second pharyngeal arches within the subset of cells that give rise to jaw primordia. Gain-of-function and loss-of-function studies in mouse, in concert with knockdown/rescue and pharmacological approaches in zebrafish, demonstrated that Ca2+ influx through CaV1.2 regulates jaw development. Cranial neural crest migration was unaffected by CaV1.2 knockdown, suggesting a role for CaV1.2 later in development. Focusing on the mandible, we observed that cellular hypertrophy and hyperplasia depended upon Ca2+ signals through CaV1.2, including those that activated the calcineurin signaling pathway. Together, these results provide new insights into the role of voltage-gated Ca2+ channels in nonexcitable cells during development.
CACNA1C 功能获得性突变的鉴定是 Timothy 综合征(TS)的病因,TS 是一种罕见的疾病,其特征是心律失常和并指畸形,这突显了 L 型电压门控钙通道 CaV1.2 在非兴奋性细胞中的意想不到的作用。异常的 Ca2+ 通过 CaV1.2 内流如何导致大多数受影响个体的表型,如伴发的并指畸形或颅面异常,这不能用已建立的 CaV1.2 作用来轻易解释。在这里,我们表明 CaV1.2 在第一和第二咽弓中表达,这些细胞是颌原基的来源。在小鼠中的功能获得和功能丧失研究,与斑马鱼中的敲低/挽救和药理学方法相结合,表明 Ca2+ 通过 CaV1.2 的内流调节颌的发育。颅神经嵴迁移不受 CaV1.2 敲低的影响,这表明 CaV1.2 在发育后期发挥作用。我们专注于下颌骨,观察到细胞肥大和增生取决于 CaV1.2 的 Ca2+ 信号,包括那些激活钙调神经磷酸酶信号通路的信号。总之,这些结果为电压门控钙通道在发育过程中非兴奋性细胞中的作用提供了新的见解。