Methods and nucleic acid molecules for aav vector selection
US-2024417717-A1 · Dec 19, 2024 · US
US9540659B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9540659-B2 |
| Application number | US-201314410983-A |
| Country | US |
| Kind code | B2 |
| Filing date | Mar 14, 2013 |
| Priority date | Jul 6, 2012 |
| Publication date | Jan 10, 2017 |
| Grant date | Jan 10, 2017 |
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An adeno-associated virus filler component comprising a nucleic acid of between 3300 and 4200 nucleotides in length is disclosed.
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What is claimed is: 1. An adeno-associated virus (AAV) filler component comprising a nucleic acid having at least 90% identity to SEQ ID NO:1 or SEQ ID NO:2, wherein the nucleic acid is between 3500 and 4000 nucleotides. 2. An adeno-associated virus (AAV) filler component consisting of a nucleic acid having at least 90% identity to SEQ ID NO:1 or SEQ ID NO:2. 3. The AAV filler component of claim 2 , wherein the nucleic acid is between 3500 and 4000 nucleotides. 4. The AAV filler component of claim 1 , wherein the nucleic acid is between 3700 and 3850 nucleotides. 5. A recombinant adeno-associated virus (AAV) vector comprising the filler component of claim 1 operably linked to an expression cassette, wherein the AAV vector is approximately 5 kb in length. 6. The AAV vector of claim 5 , wherein the expression cassette comprises a promoter. 7. The AAV vector of claim 6 , wherein the promoter is a pol III promoter. 8. The AAV vector of claim 7 , wherein the promoter is a mU6 promoter. 9. The AAV vector of claim 5 , further comprising a target sequence. 10. The AAV vector of claim 9 , wherein the target sequence is an RNAi molecule. 11. The AAV vector of claim 5 , wherein the AAV vector is an AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, or AAV8 serotype. 12. The AAV vector of claim 5 , further comprising an inverted terminal repeat (ITR) of any one of serotype AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV7, or AAV8. 13. The AAV filler component of claim 1 , wherein the nucleic acid has at least 95% identity to SEQ ID NO:1 or SEQ ID NO:2. 14. The AAV filler component of claim 1 , wherein the nucleic acid has at least 98% identity to SEQ ID NO:1 or SEQ ID NO:2. 15. The AAV filler component of claim 1 , wherein the nucleic acid has at least 99% identity to SEQ ID NO:1 or SEQ ID NO:2. 16. The AAV filler component of claim 2 , wherein the nucleic acid has at least 95% identity to SEQ ID NO:1 or SEQ ID NO:2. 17. The AAV filler component of claim 2 , wherein the nucleic acid has at least 98% identity to SEQ ID NO:1 or SEQ ID NO:2. 18. The AAV filler component of claim 2 , wherein the nucleic acid has at least 99% identity to SEQ ID NO:1 or SEQ ID NO:2.
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