SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)
First Claim
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1. A method of treating Fibrodysplasia Ossificans Progressiva (FOP) in a subject, comprising the step of administering to said subject a therapeutically effective amount of a siRNA specific against a nucleic acid encoding a mutated Activin A type I receptor (ACVR1) represented by SEQ ID NO:
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
26, wherein said siRNA has a passenger strand selected from SEQ ID NO;
56 or SEQ ID NO;
57.
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Abstract
This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to compositions and methods for siRNA-based regulation of mutated ACVR1 expression in the treatment of Fibrodysplasia Ossificans Progressiva (FOP).
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Citations
9 Claims
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1. A method of treating Fibrodysplasia Ossificans Progressiva (FOP) in a subject, comprising the step of administering to said subject a therapeutically effective amount of a siRNA specific against a nucleic acid encoding a mutated Activin A type I receptor (ACVR1) represented by SEQ ID NO:
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
26, wherein said siRNA has a passenger strand selected from SEQ ID NO;
56 or SEQ ID NO;
57. - View Dependent Claims (2, 3, 4, 5)
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
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6. A method of treating a pathology associated with heterotopic ossification in a subject, comprising the step of administering to said subject a therapeutically effective amount of a siRNA specific against a nucleic acid encoding a mutated Activin A type I receptor (ACVR1) represented by SEQ ID NO:
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
26, wherein said siRNA has a passenger strand selected from SEQ ID NO;
56 or SEQ ID NO;
57. - View Dependent Claims (7, 8)
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
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9. An allele-specific siRNA, comprising a siRNA specific against a nucleic acid encoding a mutated Activin A type I receptor (ACVR1) represented by SEQ ID NO:
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
26 wherein said siRNA has a passenger strand selected from SEQ ID NO;
56 or SEQ ID NO;
57.
- 21 relative to a nucleic acid encoding wild-type Activin A type I receptor protein (ACVR1) as set forth in SEQ ID NO;
Specification