Compositions and methods relating to myomaker-induced muscle cell fusion

Abstract

The present disclosure describes the fusogenic activity of the Myomaker protein. This polypeptide, when expressed in non-muscle cells, is able to drive fusion of the cell with a muscle cell, but not with other non-muscle cells. The use of this protein and cell expressing it in the delivery of exogenous genetic material to muscle cells also is described.

What is claimed is: 
     
         1 . A method of preparing a non-muscle cell fusion partner comprising transferring into a non-muscle cell an exogenous nucleic acid encoding a Myomaker protein under the control of a promoter active in the non-muscle cell. 
     
     
         2 . The method of  claim 1 , wherein the cell is stably transformed. 
     
     
         3 . The method of  claim 1 , wherein the cell is transiently transfected. 
     
     
         4 . The method of  claim 1 , further comprising transferring into the cell a nucleic acid encoding or sufficient to produce a detectable marker. 
     
     
         5 . The method of  claim 1 , wherein the exogenous nucleic acid is under the control of constitutive promoter. 
     
     
         6 . The method of  claim 1 , wherein the exogenous nucleic acid is under the control of an inducible promoter. 
     
     
         7 . The method of  claim 1 , wherein the non-muscle cell is an isolated cell. 
     
     
         8 . The method of  claim 1 , wherein the non-muscle cell is a human cell. 
     
     
         9 . The method of  claim 1 , wherein the non-muscle cell is a fibroblast, bone marrow cell or blood cell. 
     
     
         10 . The method of  claim 1 , where the exogenous nucleic acid further encodes a selectable marker. 
     
     
         11 . The method of  claim 1 , wherein the non-muscle cell is transformed to express a gene of interest other than Myomaker. 
     
     
         12 . The method of  claim 11 , wherein the gene of interest is a therapeutic gene. 
     
     
         13 . The method of  claim 12 , wherein the gene of interest comprises a pathologic phenotype. 
     
     
         14 . The method of  claim 13 , wherein the pathologic phenotype is selected from the group consisting of congenital myopathy, sarcopenia, amyotrophic lateral sclerosis, muscular dystrophy, Pompe disease and rhabdomyosarcoma.