Christina A. Pacak;Darin J. Falk;Lara Robert DeRuisseau;Cathryn Mah;Barry John BYRNE;David D. Fuller
发明人:
Barry John BYRNE,Darin J. Falk,Christina A. Pacak,Lara Robert DeRuisseau,Cathryn Mah,David D. Fuller
申请号：
US13527350
公开号：
US20120322861A1
申请日:
2012.06.19
申请国别(地区):
US
年份:
2012
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.
