Liver-specific constructs factor VIII expression cassettes and methods of use thereof
AssigneeSangamo Therapeutics, Inc.
InventorsBrigit E. Riley, Gary K. Lee
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Abstract
Described herein are constructs used for liver-specific expression of a transgene.
Claims26
A method of providing a protein to a subject in need thereof, the method comprising administering to the liver of the subject an AAV vector, wherein the AAV vector comprises a polynucleotide expression construct between a 5′ inverted terminal repeat (ITR) and a 3′ ITR, the polynucleotide expression construct comprising at least one insulator sequence selected from SEQ ID NOs:
- 28, 29, 30, and 38, a liver-specific enhancer sequence, a promoter sequence and a transgene, wherein the transgene encodes the protein and the protein is produced in the subject.
A method of providing a protein to a subject in need thereof, the method comprising administering to the liver of the subject an AAV vector, wherein the AAV vector comprises a polynucleotide expression construct between a 5′ inverted terminal repeat (ITR) and a 3′ ITR, the polynucleotide expression construct comprising a liver-specific enhancer sequence, a promoter sequence, an intron sequence as shown in any one of SEQ ID NO:15, 16, 17, or nucleotides 340-432 of SEQ ID NO:37 and a transgene, wherein the transgene encodes the protein and the protein is produced in the subject.
A method of providing replacement Factor VIII to a subject with hemophilia A in need thereof, the method comprising administering to the liver of the subject an AAV vector, wherein the AAV vector comprises a polynucleotide expression construct between a 5′ inverted terminal repeat (ITR) and a 3′ ITR from AAV2, wherein the polynucleotide expression construct comprises the sequence of SEQ ID NO:
- 37, and wherein the AAV vector further comprises an AAV6 capsid.
Dependencies are inferred from the claim text.