Chung Leeyup, Wang Xiaoming, Zhu Li, Towers Aaron J, Cao Xinyu, Kim Il Hwan, Jiang Yong-hui
Department of Pediatrics, Duke University School of Medicine, Durham, NC 27710, United States.
University Program in Genetics and Genomics, Duke University School of Medicine, Durham, NC 27710, United States.
Brain Res. 2015 Dec 10;1629:340-50. doi: 10.1016/j.brainres.2015.10.015. Epub 2015 Oct 17.
CYFIP1 maps to the interval between proximal breakpoint 1 (BP1) and breakpoint 2 (BP2) of chromosomal 15q11-q13 deletions that are implicated in the Angelman (AS) and Prader-Willi syndrome (PWS). There is only one breakpoint (BP3) at the distal end of deletion. CYFIP1 is deleted in AS patients with the larger class I deletion (BP1 to BP3) and the neurological presentations in these patients are more severe than that of patients with class II (BP2 to BP3) deletion. The haploinsufficiency of CYFIP1 is hypothesized to contribute to more severe clinical presentations in class I AS patients. The expression of CYFIP1 is suggested to be bi-allelic in literature but the possibility of parental origin of expression is not completely excluded. We generated and characterized Cyfip1 mutant mice. Homozygous Cyfip1 mice were early embryonic lethal. However, there was a parental origin specific effect between paternal Cyfip1 deficiency (m+/p-) and maternal deficiency (m-/p+) on both synaptic transmissions and behaviors in hippocampal CA1 synapses despite no evidence supporting the parental origin difference for the expression. Both m-/p+ and m+/p- showed the impaired input-output response and paired-pulse facilitation. While the long term-potentiation and group I mGluR mediated long term depression induced by DHPG was not different between Cyfip1 m-/p+ and m+/p- mice, the initial DHPG induced response was significantly enhanced in m-/p+ but not in m+/p- mice. m+/p- but not m-/p+ mice displayed increased freezing in cued fear conditioning and abnormal transitions in zero-maze test. The impaired synaptic transmission and behaviors in haploinsufficiency of Cyfip1 mice provide the evidence supporting the role of CYFIP1 modifying the clinical presentation of class I AS patients and in human neuropsychiatric disorders.
CYFIP1定位于15号染色体q11 - q13缺失的近端断点1(BP1)和断点2(BP2)之间的区间,该缺失与天使综合征(AS)和普拉德 - 威利综合征(PWS)有关。在缺失的远端只有一个断点(BP3)。CYFIP1在患有较大的I类缺失(BP1至BP3)的AS患者中被缺失,并且这些患者的神经学表现比患有II类(BP2至BP3)缺失的患者更严重。推测CYFIP1的单倍剂量不足导致I类AS患者出现更严重的临床表现。文献表明CYFIP1的表达是双等位基因的,但不完全排除表达的亲本来源可能性。我们构建并鉴定了Cyfip1突变小鼠。纯合的Cyfip1小鼠在胚胎早期致死。然而,尽管没有证据支持表达的亲本来源差异,但父本Cyfip1缺陷(m + / p - )和母本缺陷(m - / p + )对海马CA1突触的突触传递和行为都有亲本来源特异性效应。m - / p + 和m + / p - 都表现出输入 - 输出反应受损和双脉冲易化。虽然Cyfip1 m - / p + 和m + / p - 小鼠之间由二羟苯甘氨酸(DHPG)诱导的长时程增强和I组代谢型谷氨酸受体介导的长时程抑制没有差异,但初始DHPG诱导的反应在m - / p + 小鼠中显著增强,而在m + / p - 小鼠中没有。m + / p - 小鼠而非m - / p + 小鼠在线索性恐惧条件反射中表现出增加的僵立反应,并且在零迷宫试验中表现出异常转换。Cyfip1小鼠单倍剂量不足时突触传递和行为受损,这为支持CYFIP1在改变I类AS患者临床表现以及人类神经精神疾病中的作用提供了证据。