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.
Claims8
A method of treating Pompe disease in a human patient having Pompe disease, said method comprising administering to said patient a therapeutically effective amount of a pseudotyped recombinant adeno-associated virus (rAAV) rAAV2/9, wherein said pseudotyped rAAV comprises a nucleic acid encoding a human alpha-glucosidase (GAA) polypeptide operably linked to a promoter,
- wherein said promoter mediates expression of said GAA polypeptide in a muscle cell, wherein said promoter is selected from a desmin promoter and a creatine kinase promoter.
Dependencies are inferred from the claim text.