Methods and Gene Therapy Constructs for Treating GM2 Gangliosidoses

• 收藏
• 分享

专利权人:

发明人:

Jagdeep Singh Walia

申请号：

US15617465

公开号：

US20180028685A1

申请日:

2017.06.08

申请国别(地区):

US

年份:

2018

代理人:

摘要：

Disclosed are novel gene therapy constructs containing both HEXA and HEXB genes to treat GM2 gangliosidoses, including Sandhoff disease and Tay-Sachs disease. Also described are co-treatments using chaperone and anti-inflammatory agents to enhance the effects of gene therapy.

来源网站：

中国工程科技知识中心

来源网址：

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