Modified factor viii and factor ix genes and vectors for gene therapy
AssigneeAsklepios Biopharmaceutical, Inc.
InventorRICHARD J. SAMULSKI
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Abstract
The present invention relates to a modified and optimized Factor VIII or Factor IX nucleic acid for inclusion in a chimeric virus vector. Use of such vector can be used for treatment of hemophilia.
Claims6
A composition comprising an isolated optimized gene modified to increase CG sequences and reduce cis motifs relative to a wild type gene, wherein the optimized gene is selected from the group consisting of FVIII and FIX, wherein when the optimized gene is FVIII, the gene has the nucleotide sequence of SEQ ID NO:
- 12 or 15 or a nucleotide sequence having 95% identity thereto, and wherein when the optimized gene is FIX the gene has the nucleotide sequence of SEQ ID NO 7 or a nucleotide sequence having 95% identity thereof.
A composition comprising an optimized FVIII or FIX peptide, wherein the optimized FVIII or FIX peptide is produced by the process comprising:
- a) transfecting a host cell with an optimized FVIII or FIX gene, wherein when the optimized gene is FVIII, the gene has the nucleotide sequence of SEQ ID NO:
- 12 or 15 or a nucleotide sequence having 95% identity thereto, and wherein when the optimized gene is FIX the gene has the nucleotide sequence of SEQ ID NO 7 or a nucleotide sequence having 95% identity thereof;
- and
- b) maintaining the transformed host cell under biological conditions sufficient for expression of the peptide.
An optimized FVIII or FIX peptide, wherein the optimized FVIII or FIX peptide is encoded by an optimized FVIII or FIX gene, wherein when the optimized gene is FVIII, the gene has the nucleotide sequence of SEQ ID NO:
- 12 or 15 or a nucleotide sequence having 95% identity thereto, and wherein when the optimized gene is FIX the gene has the nucleotide sequence of SEQ ID NO 7 or a nucleotide sequence having 95% identity thereof.
Dependencies are inferred from the claim text.