Methods and nucleic acid molecules for aav vector selection
US-2024417717-A1 · Dec 19, 2024 · US
US9504762B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9504762-B2 |
| Application number | US-201414482648-A |
| Country | US |
| Kind code | B2 |
| Filing date | Sep 10, 2014 |
| Priority date | Sep 12, 2013 |
| Publication date | Nov 29, 2016 |
| Grant date | Nov 29, 2016 |
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The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
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What is claimed: 1. An adeno-associated virus (AAV) vector, comprising an AAV2 5′ inverted terminal repeat (ITR), a liver specific transcriptional regulatory region, a functionally active FVIII coding region, a polyadenylation sequence, an AAV2 3′ ITR, and optionally one or more introns, wherein the functionally active FVIII coding region comprises nucleotides 923-5296 of SEQ ID NO: 9. 2. A method of producing a recombinant adeno-associated virus (AAV) particle…
Human Necessities · mapped topic
Human Necessities · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
Chemistry & Metallurgy · mapped topic
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