HUMAN METABOLIC ENZYMES PRODUCED IN PLANTS

相当数量的遗传性代谢储存障碍 人类 有可能通过补充外源性的 生产的 酵素。葡萄糖脑苷酶，来源于人胎盘 组织 或来自中国仓鼠卵巢细胞的重组形式，目前正在 使用 一种有效但昂贵的高谢病治疗方法。我们 发现 转基因植物有能力合成 这 具有高比活力的人溶酶体酶。我们开发了 一本小说 从烟叶中提取酶的生物处理方法， 提供 至少将酶的浓度提高30倍，以使 商业化生产。通过开发一个全面的试点 制程 用于提取和纯化多克数量的 葡萄糖脑苷酶，我们将确定植物是一个重要的 低- 大量复杂重组的成本来源 蛋白质类 以推动现代医学疗法的发展。我们的目标是 减缩 高雪病患者的酶替代疗法的费用； 验证 利用植物作为重组蛋白质来源的概念 临床应用于人类；并提供准确的经济生产 型号 以评估各种未来的医疗应用。

A considerable number of hereditary metabolic storage disorders of humans are potentially treatable by supplementation with exogenously produced enzymes. Glucocerebrosidase, either derived from human placental tissue or a recombinant form from Chinese hamster ovary cell, is presently used in an effective but costly treatment of Gaucher disease. We discovered that genetically modified plants have the capacity to synthesize this human lysosomal enzyme with high specific activity. We developed a novel bioprocessing method to extract the enzyme from tobacco leaves, providing at least a 30-fold enrichment for enzyme to levels that will enable commercial production. Through development of a full-scale pilot process for the extraction and purification of multigram quantities of glucocerebrosidase, we will establish that plants are an important low- cost source for the large quantities of complex recombinant proteins required to advance modern medical therapies. Our goals are to reduce the costs of enzyme replacement therapy for Gaucher patients; validate the concept of using plants as a source of recombinant proteins for clinical use in humans; and provide an accurate economic production model to evaluate a wide variety of future medical applications.