Adeno-associated virus vectors for expression of factor VIII by target cells
AssigneeAvigen, Inc.
InventorsLinda B. Couto, Peter C. Colosi, Xiaobing Qian
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Abstract
The present invention provides improved viral vectors useful for the expression of genes at high levels in human cells. In particular, the present invention provides recombinant adeno-associated vectors (AAV) suitable for gene therapy. These vectors are capable of delivering nucleic acid containing constructs which result in the production of full-length therapeutic levels of biologically active Factor VIII in the recipient individual in vivo. The present invention also provides pharmaceutical compositions comprising such AAV vectors, as well as methods for making and using these constructs.
Claims25
A method of treating hemophilia in a mammal, comprising:
- a. providing recombinant adeno-associated virus virions comprising a nucleotide sequence encoding Factor VIII operably linked to expression control elements;
- and
- b. administering said recombinant adeno-associated virus virions to a mammal under conditions that result in the expression of Factor VIII protein at a level that provides a therapeutic effect in said mammal.
A method of treating hemophilia in a mammal, said method comprising:
- a. providing a first recombinant adeno-associated virus virion comprising a nucleotide sequence encoding the light chain of Factor VIII operably linked to expression control elements;
- and
- b. providing a second recombinant adeno-associated virus virion comprising a nucleotide sequence encoding the heavy chain of Factor VIII operably linked to expression control elements;
- and
- c. administering said first and second recombinant adeno-associated virus virions to a mammal under conditions that result in the expression of Factor VIII protein at a level that provides a therapeutic effect in said mammal.
Dependencies are inferred from the claim text.