USE OF STRUCTURAL PROTEOMIC METHODOLOGIES TOWARDS THE DEVELOPMENT OF PROTEIN THERAPEUTICS
作者单位:Department of Molecular Biology The Scripps Research Institute10550 N.Torrey Pines RdLa Jolla CA 92037 USA Department of Molecular Biology The Scripps Research Institute10550 N.Torrey Pines RdLa Jolla CA 92037 USA Department of Molecular Biology The Scripps Research Institute10550 N.Torrey Pines RdLa Jolla CA 92037 USA
会议名称:《The 4th ISGO International Conference on Structural Genomics》
会议日期:2006年
学科分类:1001[医学-基础医学(可授医学、理学学位)] 10[医学]
基 金:This work was funded by the Michaux and Piziali families MACPAD and BioMarin Pharmaceuticals
摘 要:正Employing the technologies and methodologies developed as part of the structural genomics efforts within the Stevens laboratory at TSRI, we have developed a protein therapeutic for Phenylketonuria (PKU). The disease PKU is an inherited metabolic disorder that results in phenylalanine accumulation in the body and can lead to irreversible brain damage. Initially, we analyzed all enzymes known to