US2022025335A1PendingUtilityA1

Muscular dystrophy chimeric cells and method for treating muscular dystrophies

Assignee: UNIV ILLINOISPriority: Jun 11, 2015Filed: Oct 12, 2021Published: Jan 27, 2022
Est. expiryJun 11, 2035(~8.9 yrs left)· nominal 20-yr term from priority
Inventors:Maria Siemionow
A61P 21/00C12N 5/16A61P 25/14C12N 5/0663A61K 35/34C12N 2510/00A61K 45/06C12N 5/0658A61K 9/0019A61K 35/28
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Claims

Abstract

A Muscular Dystrophy Chimeric Cell generated by ex vivo fusion of a myoblast with a second myoblast, mesenchymal stem cell, or stromal cell is described as is the use of the same in the treatment of a muscular dystrophy.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . An isolated Muscular Dystrophy Chimeric Cell (MDCC) population comprising cells produced by ex vivo fusion and expansion of
 (a) a first myoblast; and   (b) a second myoblast, mesenchymal stem cell, or stromal cell,   wherein at least one of the first myoblast, second myoblast, mesenchymal stem cell, or stromal cell is from a healthy donor and the other is a dystrophin deficient cell from subject suffering from a muscular dystrophy.   
     
     
         2 . The MDCC of  claim 1 , wherein the first myoblast and second myoblast are from different donors. 
     
     
         3 . The isolated MDCC of  claim 1 , wherein the first myoblast, second myoblast, mesenchymal stem cell, or stromal cell is autologous or allogeneic. 
     
     
         4 . The isolated MDCC of  claim 1 , wherein the healthy donor is the subject's father. 
     
     
         5 . The isolated MDCC of  claim 1 , wherein the mesenchymal stem cells are derived from bone marrow or adipose tissue. 
     
     
         6 . The isolated MDCC of  claim 1 , wherein said MDCC secretes one or more immunomodulatory cytokines and growth factors. 
     
     
         7 . The isolated MDCC of  claim 6 , wherein the one or more immunomodulatory cytokines and growth factors comprise insulin-like growth factor 1, hepatocyte growth factor and myostatin. 
     
     
         8 . The isolated MDCC of  claim 1 , wherein said an ex vivo fusion is between:
 a) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human myoblast from a healthy donor;   b) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human mesenchymal stem cell from a healthy donor; or   c) a human myoblast from a healthy donor and a dystrophin deficient human mesenchymal stem cell from a subject suffering from a muscular dystrophy; or   d) a dystrophin deficient human myoblast from a subject suffering from a muscular dystrophy and a human stromal cell from a healthy donor.   
     
     
         9 . A composition comprising the isolated MDCC of  claim 1  and a pharmaceutically acceptable carrier. 
     
     
         10 . A method of treating a muscular dystrophy comprising administering to the subject in need of treatment an effective amount of the isolated MDCC of  claim 1  thereby treating the subject's muscular dystrophy. 
     
     
         11 . The method of  claim 10 , wherein the muscular dystrophy is Duchenne Muscular Dystrophy. 
     
     
         12 . The method of  claim 10 , wherein said MDCC is administered by intravenous injection, intra-bone injection or intramuscular injection.

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