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US 2019/0194689 A1Published application
Cns targeting aav vectors and methods of use thereof
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Hongwei Zhang, Hongyan Wang, Zuoshang Xu
Life of this patent
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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.
Claims19
1-55. (canceled)
A method for delivering a transgene to CNS tissue in a subject, the method comprising:
- administering an effective amount of a recombinant adeno-associated virus (rAAV) by intracerebral injection into the putamen of the subject, wherein the rAAV comprises (i) an AAV capsid protein, and (ii) a nucleic acid comprising a transgene, wherein the transgene encodes aromatic L-amino acid decarboxylase (AADC).
A method for treating Parkinson's Disease in a subject, the method comprising:
- administering an effective amount of a recombinant adeno-associated virus (rAAV) by intracerebral injection into the putamen of the subject, wherein the rAAV. comprises (i) an AAV capsid protein, and (ii) a nucleic acid comprising a transgene, wherein the transgene encodes aromatic L-amino acid decarboxylase (AADC).
Dependencies are inferred from the claim text.