# US 2013/0156734 A1 · Compositions and Methods for Treating Friedreich's Ataxia with Interferon Gamma

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- Number: US 2013/0156734 A1
- Document ID: US20130156734A1
- Status: Published application
- Kind: A1
- Application number: 13/820,092
- Filed: 2011-08-30
- Published: 2013-06-20
- Earliest claimed: 2010-08-30
- Inventors: Roberto Testi; Barbara Tomassini
- Web page: https://patentgrep.com/patent/US20130156734A1

## CPC

- A61K 38/217: {IFN-gamma}
- A61P 25/00: Drugs for disorders of the nervous system
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS

## Abstract

Described herein are compositions and methods for treating Friedreich's Ataxia (FRDA) with interferon gamma. In some aspects, methods for increasing expression of frataxin in cells and for treating Friedreich's Ataxia are provided. In some additional aspects, methods increasing aconitase activity in cells are provided.

## Claims

### Claim 1

A method of increasing expression of frataxin in a cell identified as having a deficient amount of frataxin or as having Friedrich's Ataxia, comprising administering an effective amount of interferon gamma to said cell.

### Claim 2

(canceled)

### Claim 3

A method of treating Friedrich's Ataxia, comprising administering to a subject diagnosed as having Friedrich's Ataxia a therapeutically effective amount of interferon gamma.

### Claim 4

A method of upregulating frataxin, comprising administering to a subject in need thereof a therapeutically effective amount of interferon gamma.

### Claim 5 (depends on 3)

The method of claim 3, wherein said interferon gamma is recombinant.

### Claim 6 (depends on 5)

The method of claim 5, wherein said recombinant interferon gamma is ACTIMMUNE™.

### Claim 7 (depends on 3)

The method of claim 3 wherein said interferon gamma consists or comprises of any one of the sequences in Table 2.

### Claim 8 (depends on 3)

The method of claim 3 wherein said interferon gamma consists or comprises of SEQ ID NO: 2.

### Claim at position 9

-13. (canceled)

### Claim 14 (depends on 4)

The method of claim 4, wherein said interferon gamma is recombinant.

### Claim 15 (depends on 14)

The method of claim 14, wherein said recombinant interferon gamma is ACTIMMUNE™.

### Claim 16 (depends on 4)

The method of claim 4 wherein said interferon gamma consists or comprises of SEQ ID NO: 2.

### Claim 17 (depends on 4)

The method of claim 4 wherein said interferon gamma consists or comprises of any one of the sequences in Table 2.