Fromme J Christopher, Ravazzola Mariella, Hamamoto Susan, Al-Balwi Mohammed, Eyaid Wafaa, Boyadjiev Simeon A, Cosson Pierre, Schekman Randy, Orci Lelio
Department of Molecular and Cell Biology, University of California, Berkeley, Berkeley, CA 94720, USA.
Department of Cell Physiology and Metabolism, University of Geneva Medical Center, 1211 Geneva 4, Switzerland.
Dev Cell. 2007 Nov;13(5):623-634. doi: 10.1016/j.devcel.2007.10.005.
Proteins trafficking through the secretory pathway must first exit the endoplasmic reticulum (ER) through membrane vesicles created and regulated by the COPII coat protein complex. Cranio-lenticulo-sutural dysplasia (CLSD) was recently shown to be caused by a missense mutation in SEC23A, a gene encoding one of two paralogous COPII coat proteins. We now elucidate the molecular mechanism underlying this disease. In vitro assays reveal that the mutant form of SEC23A poorly recruits the Sec13-Sec31 complex, inhibiting vesicle formation. Surprisingly, this effect is modulated by the Sar1 GTPase paralog used in the reaction, indicating distinct affinities of the two human Sar1 paralogs for the Sec13-Sec31 complex. Patient cells accumulate numerous tubular cargo-containing ER exit sites devoid of observable membrane coat, likely representing an intermediate step in COPII vesicle formation. Our results indicate that the Sar1-Sec23-Sec24 prebudding complex is sufficient to form cargo-containing tubules in vivo, whereas the Sec13-Sec31 complex is required for membrane fission.
通过分泌途径运输的蛋白质必须首先通过由COPII衣被蛋白复合物产生和调节的膜泡离开内质网(ER)。颅-晶状体-缝合发育异常(CLSD)最近被证明是由SEC23A中的一个错义突变引起的,SEC23A是编码两种同源COPII衣被蛋白之一的基因。我们现在阐明了这种疾病的分子机制。体外实验表明,SEC23A的突变形式难以募集Sec13-Sec31复合物,从而抑制囊泡形成。令人惊讶的是,这种效应受到反应中使用的Sar1 GTP酶同源物的调节,这表明两个人类Sar1同源物对Sec13-Sec31复合物具有不同的亲和力。患者细胞积累了大量含有管状货物的内质网出口位点,没有可观察到的膜衣,这可能代表了COPII囊泡形成的一个中间步骤。我们的结果表明,Sar1-Sec23-Sec24预芽复合物足以在体内形成含有货物的小管,而Sec13-Sec31复合物是膜裂变所必需的。