Adeno-associated vector compositions for expression of factor VIII
OwnerLinda B. Couto, Peter C. Colosi +1inventors
InventorsLinda B. Couto, Peter C. Colosi, Xiaobing Qian
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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.
Claims1
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.
Dependencies are inferred from the claim text.