Gene therapy composition for use in diabetes treatment

Abstract

New gene therapy constructions and compositions are the subject of present invention. The gene therapy compositions consist in adeno-associated vectors which jointly express insulin (Ins) and glucokinase (Gck) genes. The new gene therapy constructions are useful for treatment of diabetes either in dosgs or human beings.

1 . Gene therapy composition which comprises a single vector carrying and allowing the expression of both an insulin gene (Ins) and a glucokinase gene (Gck) operatively linked, wherein the vector contains a coding sequence of the insulin gene and a coding sequence of the glucokinase gene that are mutated with the purpose of increasing protein production in a subject and in such a way that the codon adaptation index of these mutated coding sequences is higher than 0.9. 
     
     
         2 . The gene therapy composition of  claim 1 , wherein the vector is an adeno associated virus based vector. 
     
     
         3 . The gene therapy composition of  claim 1 , wherein the vector is an adeno associated virus vector of serotype 1 (AAV1). 
     
     
         4 . The gene therapy composition of  claim 1 , wherein the insulin gene encodes for human insulin and the glucokinase gene encodes for human glucokinase.