Fabry's disease gene therapy

• 收藏
• 分享

专利权人:

UCL Business PLC

发明人:

NATHWANI, Amit,RAJ, Deepak

申请号：

DK16721916

公开号：

DK3244931T3

申请日:

2016.05.10

申请国别(地区):

DK

年份:

2019

代理人:

摘要：

There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional ±-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule； and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.

来源网站：

中国工程科技知识中心

来源网址：

http://www.ckcest.cn/home/