Adeno-associated vector compositions for expression of factor VIII

Abstract

One form of a composition has two types of recombinant adeno-associated virus. The first type encodes a portion of Factor VIII operably linked to an expression control element; and the second type encodes a different portion of Factor VIII operably linked to an expression control element. The first and second nucleotide sequences collectively encode a functional Factor VIII protein. Another form of the composition is a recombinant adeno-associated virus containing a nucleotide sequence encoding functional Factor VIII light or heavy chain operably linked to a tissue-specific promoter.

What is claimed is:  
     
         1 . A method of treating hemophilia in a human comprsing provding at least one recombinant adeno-associated virion comprising nucleotide sequences encoding Factor VIII and administering said at least one recombinant adeno-associated virion into said human under conditions such that said Factor VIII nucleotide sequences are expressed at a level which provides a therapeutically effective amount in said human.