首页> 外国专利> Method for improving impaired Integrity of the neuromuscular Junction comprising Administration of a composition comprising a recombinant adeno-associated viral vector (rAAV) comprising a Nucleic Acid Molecule that encodes The Acid Alpha glucosidase (GAA).

Method for improving impaired Integrity of the neuromuscular Junction comprising Administration of a composition comprising a recombinant adeno-associated viral vector (rAAV) comprising a Nucleic Acid Molecule that encodes The Acid Alpha glucosidase (GAA).

机译：改善神经肌肉接头完整性受损的方法，包括施用包含重组腺相关病毒载体（rAAV）的组合物，所述重组腺相关病毒载体包含编码酸性α-葡糖苷酶（GAA）的核酸分子。

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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.

机译：本发明提供了与rAAV介导的用于治疗疾病如庞贝病的治疗有效分子的递送的组合物和使用方法。这些组合物与各种给药途径和方法相结合导致治疗分子在特定器官，组织和细胞中靶向表达。

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公开/公告号CL2014003464A1

专利类型
公开/公告日2015-06-12

原文格式PDF
申请/专利权人 UNIVERSITY OF FLORIDA RESEARCH FOUNDATION INC.;
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申请/专利号CL20140003464
发明设计人 BYRNE BARRY JOHN;FALK DARIN J-;PACAK CHRISTINA;DERUISSEAU LARA ROBERT;MAH CATHRYN;FULLER DAVID D.;
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申请日2014-12-19
分类号A61K48;A61P21;A61P21/04;
国家 CL
入库时间 2022-08-21 15:15:54

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