Factor viii sequences
US-2017049859-A1 · Feb 23, 2017 · US
US10124041B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10124041-B2 |
| Application number | US-201615254984-A |
| Country | US |
| Kind code | B2 |
| Filing date | Sep 1, 2016 |
| Priority date | Jun 12, 2012 |
| Publication date | Nov 13, 2018 |
| Grant date | Nov 13, 2018 |
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There is provided a nucleic acid molecule comprising a nucleotide sequence encoding a factor VIII protein, wherein a B domain portion of the factor VIII protein is encoded by a nucleotides sequence between 90 and 111 nucleotides in length and has an amino acid sequence that is at least 85% identical to SEQ ID NO: 4 which comprises six asparagine residues. Also provided is a factor VIII protein, a vector comprising the above nucleic acid molecule, a host cell, a transgenic animal, a method of treating Haemophilia A, and a method for the preparation of a parvoviral gene delivery vector.
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The invention claimed is: 1. A method for administering to a subject a nucleic acid molecule encoding a Factor VIII protein, comprising: intravenously administering to the subject a nucleic acid molecule encoding a Factor VIII protein, wherein the Factor VIII protein B domain comprises a spacer that is replaced and wherein the B domain comprises the amino acid sequence set forth in SEQ ID NO:4. 2. The method of claim 1 , wherein the B domain of said Factor VIII protein is between 30 and 37 amino acids in length. 3. The method of claim 1 , wherein the B domain of said Factor VIII protein is 31 amino acids in length. 4. The method of claim 1 , wherein the B domain of said Factor VIII protein comprises the amino acid sequence of SEQ ID NO:5. 5. The method of claim 1 , wherein the B domain of said Factor VIII protein consists of the amino acid sequence of SEQ ID NO:5. 6. The method of claim 1 , wherein the Factor VIII protein encoded by the nucleic acid molecule comprises domains A1, A2, A3, C1 and C2, and wherein the nucleic acid molecule is codon optimised compared to a corresponding wild type sequence. 7. The method of claim 1 , wherein said Factor VIII protein comprises the amino acid sequence of SEQ ID NO:6. 8. The method of claim 1 , wherein said nucleic acid molecule encoding said Factor VIII protein comprises the nucleic acid sequence of SEQ ID NO:3.
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