Methods for treating pompe disease
First Claim
1. A method for treating pompe disease in a subject comprising administering to the subject a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
2 Assignments
0 Petitions
Accused Products
Abstract
The present invention provides methods for treating Pompe disease in a subject by administering to the subject a therapeutically effective amount of a fusion protein which includes human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain. The lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
-
Citations
40 Claims
- 1. A method for treating pompe disease in a subject comprising administering to the subject a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
- 10. A method for treating Pompe disease in a subject comprising administering to the subject a therapeutically effective amount of a fusion protein comprising amino acids 1 and 8-67 of mature human insulin-like growth factor II (IGF-II) and amino acids 70-952 of human acid alpha-glucosidase (GAA).
- 14. A method for reducing glycogen levels in vivo comprising administering to a subject suffering from Pompe disease an effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
- 23. A method for reducing glycogen levels in a mammalian lysosome comprising targeting to the lysosome an effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
- 27. A method for reducing glycogen levels in a muscle tissue of a subject suffering from Pompe disease comprising delivering to the muscle tissue a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
-
29. A method for treating cardiomyopathy associated with Pompe disease in a subject comprising administering to the subject a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
-
30. A method for treating myopathy associated with Pompe disease in a subject comprising administering to the subject a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
-
31. A method for increasing acid alpha-glucosidase (GAA) activity in a subject suffering from Pompe disease comprising administering to the subject a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
- 32. A pharmaceutical composition suitable for treatment of Pompe disease comprising a therapeutically effective amount of a fusion protein comprising human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain, wherein the lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
Specification