Methods and nucleic acid molecules for aav vector selection
US-2024417717-A1 · Dec 19, 2024 · US
US9284357B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9284357-B2 |
| Application number | US-201414246560-A |
| Country | US |
| Kind code | B2 |
| Filing date | Apr 7, 2014 |
| Priority date | May 28, 2009 |
| Publication date | Mar 15, 2016 |
| Grant date | Mar 15, 2016 |
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The invention in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the invention relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.
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What is claimed is: 1. A composition comprising an isolated adeno-associated virus (AAV) vector wherein the isolated adeno-associated virus (AAV) vector comprises (i) the isolated adeno-associated virus (AAV) capsid protein of the amino acid sequence SEQ ID NO: 91; and (ii) a nucleic acid encoding a heterologous transgene. 2. The composition of claim 1 , further comprising a pharmaceutically acceptable carrier. 3. The composition of claim 1 , wherein the heterologous transgene comprises a tissue-specific promoter operably linked to a coding sequence. 4. The composition of claim 3 , wherein the coding sequence encodes a therapeutic protein. 5. The composition of claim 4 , wherein the therapeutic protein is aromatic amino acid decarboxylase (AADC).
Drugs for disorders of the cardiovascular system · CPC title
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Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy · CPC title
Drugs for disorders of the respiratory system · CPC title
Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00) · CPC title
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