Compositions and methods for treating diseases
AssigneeUniversity of Florida Research Foundation, Inc.
InventorsBarry John Byrne, Darin J. Falk, Christina Pacak, Lara Robert DeRuisseau, Cathryn Mah, David D. Fuller
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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.
Claims3
A method of treating Pompe disease in a human patient suffering therefrom, said method comprising administering to said patient a therapeutically effective amount of a pseudotyped recombinant adeno-associated virus (rAAV) selected from the group consisting of:
- rAAV2/8 and rAAV2/9, wherein said rAAV comprises a nucleic acid encoding a human alpha-glucosidase (GAA) polypeptide operably linked to a desmin promoter, wherein said rAAV is administered to said patient intravenously, intrathecally, intraspinally, and/or intramuscularly.
Dependencies are inferred from the claim text.