THERAPEUTICS OF HUMAN PROTEINS PRODUCED IN PLANTS

人血清蛋白α 1-抗胰蛋白酶（AAT）用于治疗 肺气肿，但目前的生产不能满足需求。酵母- 从血清中清除注射到大鼠中的衍生重组AAT 太快而不能产生治疗效果。这里的目的是测试 转基因植物是否可以用来生产AAT或其他蛋白质 对人类有治疗作用的药物。一旦证明了这一点， 将导致AAT生产和使用的突破， 治疗肺气肿和许多其他炎症相关疾病。 转基因水稻种子的发芽将使AAT的生产在广大 数量和单位成本低。 第一阶段的具体目标是从转基因植物中纯化AAT蛋白， 水稻细胞系，表征AAT蛋白结构，并测试 注射大鼠后AAT的清除时间。的结果 第一阶段将确定转基因植物衍生的AAT是否具有 适当的结构，从而在大鼠中提供更长的清除时间， 比酵母衍生的AAT具有更大的治疗潜力。 拟定商业应用： 在水稻植物细胞中表达人蛋白质提供了低成本， 可能具有治疗价值的蛋白质的高容量来源。

The human serum protein alpha1-Antitrypsin (AAT) is used for treatment of emphysema, but current production cannot satisfy the demand. Yeast- derived recombinant AAT injected into the rat was cleared from the serum too rapidly to have therapeutic benefit. The objective here is to test whether transgenic plants can be used to produce AAT or other proteins that have therapeutic benefits in humans. Once this is demonstrated, it will lead to a breakthrough in the production and use of AAT for treatment of emphysema and many other inflammation-related disorders. Malting of transgenic rice seeds will allow production of AAT in vast quantities and at low cost per unit. The specific aims in Phase I are to purify AAT protein from transgenic rice cell lines, to characterize the AAT protein structure and to test the clearing time of this AAT after injection into rats. The results of phase I will determine whether transgenic plant-derived AAT has the proper structure, thus providing a longer clearing time in rats and greater therapeutic potential than yeast-derived AAT. PROPOSED COMMERCIAL APPLICATION: Expression of human proteins in rice plant cells provided a low cost, high volume source of proteins that may have therapeutic value.