Methods and nucleic acid molecules for aav vector selection
US-2024417717-A1 · Dec 19, 2024 · US
US9102949B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9102949-B2 |
| Application number | US-201113642719-A |
| Country | US |
| Kind code | B2 |
| Filing date | Apr 22, 2011 |
| Priority date | Apr 23, 2010 |
| Publication date | Aug 11, 2015 |
| Grant date | Aug 11, 2015 |
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The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
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What is claimed is: 1. A method for treating Canavan disease in a subject, the method comprising: intrathecally, intraventricularly, or intravascularly administering rAAV to the subject in an amount effective for transducing oligodendrocytes of the subject with the rAAV, wherein the rAAV comprises (i) a capsid protein having the amino acid sequence of SEQ ID NO: 9 and (ii) a nucleic acid comprising a promoter operably linked with a region encoding aspartoacylase (ASPA), wherein…
Human Necessities · mapped topic
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