申请人:——
公开号:US20020088024A1
公开(公告)日:2002-07-04
The invention relates to -galactosidase truncated at the carboxy terminus and the production of enzymatically active recombinant human and animal lysosomal enzymes involving construction and expression of recombinant expression constructs comprising coding sequences of human or animal lysosomal enzymes in a plant expression system. The plant expression system provides for post-translational modification and processing to produce a recombinant gene product exhibiting enzymatic activity. The invention is demonstrated by working examples in which transgenic tobacco plants express recombinant expression constructs comprising human glucocerebrosidase nucleotide sequences. The invention is also demonstrated by working examples in which transfected tobacco plants express recombinant viral expression constructs comprising human &agr; galactosidase nucleotide sequences. The recombinant lysosomal enzymes produced in accordance with the invention may be used for a variety of purposes, including but not limited to enzyme replacement therapy for the therapeutic treatment of human and animal lysosomal storage diseases.
本发明涉及在羧基末端截短的-半乳糖苷酶,以及具有酶活性的重组人和动物溶酶体酶的生产,包括在植物表达系统中构建和表达重组表达构建体,该构建体包含人或动物溶酶体酶的编码序列。植物表达系统可进行翻译后修饰和加工,以产生具有酶活性的重组基因产物。本发明通过转基因烟草植物表达包含人葡萄糖脑苷脂核苷酸序列的重组表达构建体的工作实例来证明。本发明还通过转染烟草植物表达包含人半乳糖苷酶核苷酸序列的重组病毒表达构建体的工作实例来证明。根据本发明生产的重组溶酶体酶可用于多种目的,包括但不限于用于治疗人类和动物溶酶体贮积疾病的酶替代疗法。