Department of Pharmacology, Graduate School of Pharmaceutical Sciences, Tohoku University, Sendai 980-8578, Japan.
J Neurosci. 2011 Jan 5;31(1):346-58. doi: 10.1523/JNEUROSCI.4816-10.2011.
In humans, mutations in the gene encoding ATRX, a chromatin remodeling protein of the sucrose-nonfermenting 2 family, cause several mental retardation disorders, including α-thalassemia X-linked mental retardation syndrome. We generated ATRX mutant mice lacking exon 2 (ATRX(ΔE2) mice), a mutation that mimics exon 2 mutations seen in human patients and associated with milder forms of retardation. ATRX(ΔE2) mice exhibited abnormal dendritic spine formation in the medial prefrontal cortex (mPFC). Consistent with other mouse models of mental retardation, ATRX(ΔE2) mice exhibited longer and thinner dendritic spines compared with wild-type mice without changes in spine number. Interestingly, aberrant increased calcium/calmodulin-dependent protein kinase II (CaMKII) activity was observed in the mPFC of ATRX(ΔE2) mice. Increased CaMKII autophosphorylation and activity were associated with increased phosphorylation of the Rac1-guanine nucleotide exchange factors (GEFs) T-cell lymphoma invasion and metastasis 1 (Tiam1) and kalirin-7, known substrates of CaMKII. We confirmed increased phosphorylation of p21-activated kinases (PAKs) in mPFC extracts. Furthermore, reduced protein expression and activity of protein phosphatase 1 (PP1) was evident in the mPFC of ATRX(ΔE2) mice. In cultured cortical neurons, PP1 inhibition by okadaic acid increased CaMKII-dependent Tiam1 and kalirin-7 phosphorylation. Together, our data strongly suggest that aberrant CaMKII activation likely mediates abnormal spine formation in the mPFC. Such morphological changes plus elevated Rac1-GEF/PAK signaling seen in ATRX(ΔE2) mice may contribute to mental retardation syndromes seen in human patients.
在人类中,编码 ATRX 的基因突变,一种蔗糖非发酵 2 家族的染色质重塑蛋白,导致几种智力迟钝障碍,包括α-地中海贫血伴 X 连锁智力迟钝综合征。我们生成了缺失外显子 2 的 ATRX 突变小鼠(ATRX(ΔE2) 小鼠),这种突变模拟了人类患者中所见的外显子 2 突变,并与更温和的智力迟钝形式相关。ATRX(ΔE2) 小鼠在前扣带皮层(mPFC)中表现出异常的树突棘形成。与其他智力迟钝的小鼠模型一致,ATRX(ΔE2) 小鼠的树突棘比野生型小鼠更长更细,而树突棘数量没有变化。有趣的是,在 ATRX(ΔE2) 小鼠的 mPFC 中观察到异常增加的钙/钙调蛋白依赖性蛋白激酶 II(CaMKII)活性。CaMKII 的过度自磷酸化和活性增加与 Rac1-鸟嘌呤核苷酸交换因子(GEFs)T 细胞淋巴瘤侵袭和转移 1(Tiam1)和 kalirin-7 的磷酸化增加有关,这是 CaMKII 的已知底物。我们证实 mPFC 提取物中的 p21 激活激酶(PAKs)磷酸化增加。此外,在 ATRX(ΔE2) 小鼠的 mPFC 中,蛋白磷酸酶 1(PP1)的蛋白表达和活性降低。在培养的皮质神经元中,通过 okadaic 酸抑制 PP1 增加了 CaMKII 依赖性 Tiam1 和 kalirin-7 磷酸化。总之,我们的数据强烈表明,异常的 CaMKII 激活可能介导 mPFC 中异常的棘形成。在 ATRX(ΔE2) 小鼠中看到的这种形态变化加上 Rac1-GEF/PAK 信号的升高可能导致人类患者中出现智力迟钝综合征。