MATERIALS AND METHODS FOR TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) OR OMENN SYNDROME

• 收藏
• 分享

专利权人:

发明人:

申请号：

EP17716980.2

公开号：

EP3411078A1

申请日:

2017.02.02

申请国别(地区):

EP

年份:

2018

代理人:

摘要：

The present application provides materials and methods for treating a patient with severe combined immunodeficiency (SCID) or Omenn Syndrome, both ex vive and in vive. In addition, the present application provides materials and methods for editing to modulate the expression, function or activity of the Recombination Activating Gene 1 (RAG1) gene in a cell by genome editing.

来源网站：

中国工程科技知识中心

来源网址：

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