Optimised Coding Sequence and Promoter
AssigneesUCL Business PLC, Thrombosis Research Institute, St. Jude Children's Research Hospital
InventorsAmit Nathwani, Natalie Ward, Adrian Thrasher, Edward Tuddenham, John McVey, John Gray, Andrew Davidoff
Life of this patent
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Abstract
An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes arc alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
Claims8
1-22. (canceled)
A recombinant adeno-associated virus (AAV) particle comprising a heterologous nucleic acid sequence and a promoter that is operably linked to and drives expression of said heterologous nucleic acid sequence, wherein said promoter has at least 90% sequence identity to the nucleotide sequence of SEQ ID NO:3.
The recombinant AAV particle of claim 1, wherein said promoter is less than 350 base pairs in length.
The recombinant AAV particle of claim 1, wherein said promoter comprises the nucleotide sequence of SEQ ID NO:3.
The recombinant AAV particle of claim 1, where said promoter consists essentially of the nucleotide sequence of SEQ ID NO:3.
The recombinant AAV particle of claim 1, where said promoter consists of the nucleotide sequence of SEQ ID NO:3.
The recombinant AAV particle of claim 1 which is of AAVS serotype.
A composition of matter comprising the recombinant AAV particle of claim 1 and a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.