CRYSTALLOGRAPHIC STUDIES ON HUMAN PROTECTIVE PROTEIN

人类溶酶体积症， 其特征在于β-半乳糖苷酶的联合缺乏， 神经氨酸酶活性。 临床症状包括 神经系统、骨骼和眼睛异常， 精神发育迟滞和早期死亡例。 已经确定 这种疾病的主要缺陷实际上是缺乏 第三种溶酶体蛋白，“人类保护蛋白”-- HPP。 的 保护性蛋白质被认为与多酶复合物有关 与β-半乳糖苷酶和神经氨酸酶，防止降解， 前者并提供后者的稳定/激活， 溶酶体的酸性环境。 我们的目标是 确定前体和成熟体的三维结构 保护性蛋白的形式，通过X射线衍射分析， 蛋白质晶体

The human lysosomal storage disease galactosialidosis has been characterized as a combined deficiency of b-galactosidase and neuraminidase enzymatic activities. Clinical symptoms include neurological-, skeletal- and eye abnormalities, in the more serious cases mental retardation and early death. It has been established that the primary defect in this disease is in fact the deficiency of a third lysosomal protein, the "Human Protective Protein" - HPP. The protective protein is thought to be involved in a multi-enzyme complex with b-galactosidase and neuraminidase, preventing the degradation of the former and providing stabilization/activation of the latter, in the harsh acidic environment of the lysosome. Our goal is to determine the 3-dimensional structures of the precursor and mature forms of the protective protein, by X-ray diffraction analysis of protein crystals.