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公开(公告)号：US20180051299A9
公开(公告)日：2018-02-22
申请号：US14994477
申请日：2016-01-13
Inventor： Barry John Byrne , Darin J. Falk , Christina Pacak , Lara Robert Deruisseau , Cathryn Mah , David D. Fuller
CPC classification number: C12N15/86 , A61K9/0019 , A61K45/06 , A61K48/0058 , A61K48/0075 , C12N7/00 , C12N2710/16644 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/6027 , C12N2830/008 , C12Y302/0102
Abstract: 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.
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公开(公告)号：US20170028002A1
公开(公告)日：2017-02-02
申请号：US15088167
申请日：2016-04-01
Inventor： Barry John Byrne , Darin J. Falk , Christina Pacak , Lara Robert Deruisseau , Cathryn Mah , David D. Fuller
CPC classification number: A61K35/76 , A61K9/0019 , A61K48/005 , C12N7/00 , C12N9/2408 , C12N15/86 , C12N2710/16644 , C12N2750/14111 , C12N2750/14143 , C12N2750/14152 , C12Y302/0102
Abstract: 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.
Abstract translation: 本发明提供了用于治疗有效分子的rAAV介导递送用于治疗疾病如庞培病的组合物和方法。 这些组合物与各种途径和施用方法组合导致治疗分子在特定器官，组织和细胞中的靶向表达。
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公开(公告)号：US20160369297A1
公开(公告)日：2016-12-22
申请号：US14994477
申请日：2016-01-13
Inventor： Barry John Byrne , Darin J. Falk , Christina Pacak , Lara Robert Deruisseau , Cathryn Mah , David D. Fuller
CPC classification number: C12N15/86 , A61K9/0019 , A61K45/06 , A61K48/0058 , A61K48/0075 , C12N7/00 , C12N2710/16644 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/6027 , C12N2830/008 , C12Y302/0102
Abstract: 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.
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公开(公告)号：US20150196671A1
公开(公告)日：2015-07-16
申请号：US14409643
申请日：2013-06-19
Inventor： Barry John Byrne , Darin J. Falk , Christina Pacak , Lara Robert Deruisseau , Cathryn Mah , David D. Fuller
CPC classification number: C12N15/86 , A61K9/0019 , A61K45/06 , A61K48/0058 , A61K48/0075 , C12N7/00 , C12N2710/16644 , C12N2750/14143 , C12N2750/14145 , C12N2750/14151 , C12N2810/6027 , C12N2830/008 , C12Y302/0102
Abstract: 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.
Abstract translation: 本发明提供了用于治疗有效分子的rAAV介导递送用于治疗疾病如庞培病的组合物和方法。 这些组合物与各种途径和施用方法组合导致治疗分子在特定器官，组织和细胞中的靶向表达。
