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US 9,102,949 B2Granted
CNS targeting AAV vectors and methods of use thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Hongwei Zhang, Hongyan Wang, Zuoshang Xu
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Abstract
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.
Claims9
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 the ASPA is expressed from the nucleic acid in oligodendrocytes transduced by the rAAV.
1 more element
Dependencies are inferred from the claim text.