# US 8,771,965 B2 · Methods and compositions involving nucleotide repeat disorders

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- Number: US 8,771,965 B2
- Document ID: US8771965B2
- Status: Granted
- Kind: B2
- Application number: 13/419,475
- Filed: 2012-03-14
- Published: 2012-07-05
- Granted: 2014-07-08
- Earliest claimed: 2006-04-11
- Assignees: Board of Regents, The University of Texas System
- Applicants: Partha Sarkar; Tetsuo Ashizawa; Weidong Xu
- Inventors: Partha Sarkar; Tetsuo Ashizawa; Weidong Xu
- Web page: https://patentgrep.com/patent/US8771965B2

## CPC

- C12Q 1/6883: for diseases caused by alterations of genetic material
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61P 25/28: for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A61P 31/00: Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P 35/00: Antineoplastic agents
- C12Q 1/6897: involving reporter genes operably linked to promoters
- G01N 33/6887: {from muscle, cartilage or connective tissue}
- C12Q 2600/158: Expression markers
- G01N 2500/02: Screening involving studying the effect of compounds C on the interaction between interacting molecules A and B (e.g. A = enzyme and B = substrate for A, or A = receptor and B = ligand for the receptor)
- C12Q: MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS (immunoassay G01N33/53); COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- G01N: INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES (measuring or testing apparatus or processes other than immunoassay, involving enzymes or microorganisms C12M, C12Q)

## Abstract

The present invention concerns the methods and compositions involving nucleic acids with long repeat sequences. In some embodiments of the invention, there are methods for generating such a nucleic acid, and in other methods, there are methods for using such a nucleic acid to screen for candidate therapeutic compounds. Furthermore the present invention relates to methods of screening for Notch inhibitors and other substances that may be used to treat muscle loss and wasting.

## Claims

### Claim 1

A method of treating muscle loss or muscle wasting in a patient comprising administering skeletrophin to the patient, wherein skeletrophin has the amino acid sequence of SEQ ID NO:4.

### Claim 2 (depends on 1)

The method of claim 1, wherein skeletrophin is provided by administering a nucleic acid encoding the skeletrophin protein.

### Claim 3 (depends on 2)

The method of claim 2, wherein the nucleic acid is comprised in a vector.

### Claim 4 (depends on 1)

The method of claim 1, wherein the vector is a viral vector.

### Claim 5 (depends on 1)

The method of claim 1, wherein skeletrophin or a nucleic acid encoding skeletrophin is administered to the patient intramuscularly, orally, intravenously, or subcutaneously.