Materials and methods for treating Friedreich's Ataxia
AssigneeThe Trustees of Indiana University
InventorRonald Mark Payne
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Abstract
A TAT-FXN fusion polypeptide useful in treating subjects diagnosed with Friedrich's Ataxia, hypertrophic cardiomyopathy, or both are disclosed, as are related methods of treatment and pharmaceutical compositions.
Claims18
A method of treating Friedreich's Ataxia (FRDA), said method comprising administering to a subject in need thereof a fusion polypeptide consisting of:
- a first peptide having an amino acid sequence of SEQ ID NO:
- 2;
- a second peptide having an amino acid sequence of SEQ ID NO:
- 4;
- and
- a 2-amino acid linker disposed between the first and second peptides.
A method of treating Friedreich's Ataxia (FRDA), said method comprising administering to a subject in need thereof a fusion polypeptide consisting of the amino acid sequence of SEQ ID NO:
- 1.
A method of treating Friedreich's Ataxia (FRDA), said method comprising administering to a subject in need thereof a fusion polypeptide comprising:
- a first peptide having an amino acid sequence of SEQ ID NO:
- 2;
- and
- a second peptide having an amino acid sequence of SEQ ID NO:
- 4;
- wherein the first peptide is fused through a 2-amino acid linker to the second peptide.
A method of treating Friedreich's Ataxia (FRDA), said method comprising administering to a subject in need thereof a fusion polypeptide comprising the amino acid sequence of SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.