COMPOSITIONS AND METHODS FOR TREATING DISEASES

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专利权人:

University of Florida Research Foundation; Inc.

发明人:

BYRNE, Barry, John,FALK, Darin, J.,PACAK, Christina,Deruisseau, Lara, Robert,MAH, Cathryn,FULLER, David, D.

申请号：

DK17184780

公开号：

DK3292875T3

申请日:

2013.06.19

申请国别(地区):

DK

年份:

2020

代理人:

摘要：

The present invention provides compositions and methods of use pertaining to rAAV- mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

来源网站：

中国工程科技知识中心

来源网址：

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