# US 11,306,287 B1 · Method of generating skeletal muscle stem cells from pluripotent cells

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- Number: US 11,306,287 B1
- Document ID: US11306287B1
- Status: Granted
- Kind: B1
- Application number: 15/625,619
- Filed: 2017-06-16
- Granted: 2022-04-19
- Earliest claimed: 2016-06-16
- Assignees: Regents of the University of Minnesota
- Applicants: Regents of the University of Minnesota
- Inventors: Michael Kyba; Sun Kin Sunny Chan; Robert W. Arpke
- Web page: https://patentgrep.com/patent/US11306287B1

## CPC

- C12N 5/0607: {Non-embryonic pluripotent stem cells, e.g. MASC (induced pluripotent stem cells C12N5/0696)}
- C12N 5/0658: {Skeletal muscle cells, e.g. myocytes, myotubes, myoblasts}
- A61K 35/34: Muscles; Smooth muscle cells; Heart; Cardiac stem cells; Myoblasts; Myocytes; Cardiomyocytes (vascular smooth muscle A61K35/44)
- A61P 21/00: Drugs for disorders of the muscular or neuromuscular system
- A61K 48/00: Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- C12N 2501/105: Insulin-like growth factors [IGF]
- C12N 2510/00: Genetically modified cells
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- 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

Compositions and methods of the invention are directed to the formation and use of teratoma-derived skeletal muscle stem cells. In one embodiment, the teratoma-derived skeletal muscle stem cells improve muscle contractile force (definition “functional”) when administered to a patient in need thereof.

## Claims

### Claim 1

An isolated skeletal muscle stem cell that does not comprise a genetic modification of Pax3 and wherein 40,000 of said stem cells, upon injection into a tibialis anterior muscle, can reconstitute 70-80% of said muscle volume and is produced by:
a) injecting pluripotent stem cells into an immunodeficient animal,
b) allowing one or more teratomas to form in said animal of a), and
c) isolating said skeletal muscle stem cells from the one or more teratomas of b).

### Claim 2

Isolated teratoma derived skeletal muscle stem cells that are Lin− α7-integrin+ VCAM1+(α7+ VCAM) and have no genetic modification of Pax3, wherein 40,000 of said stem cells, upon injection into a tibialis anterior muscle can reconstitute 70-80% of said muscle volume.

### Claim 3

Isolated teratoma derived skeletal muscle stem cells that are CD45-neg CD31-neg VCAM1+Itga7+ or CD45-neg CD31-neg CD56+ and have no genetic modification of Pax3, wherein 40,000 of said stem cells, upon injection into a tibialis anterior muscle can reconstitute 70-80% of said muscle volume.

### Claim 4

Isolated teratoma derived skeletal muscle stem cells that are CD9+, CD29+, CD46+, CD73−, CD82+ or a combination thereof and have no genetic modification of Pax3, wherein 40,000 of said stem cells, upon injection into a tibialis anterior muscle can reconstitute 70-80% of said muscle volume.
