Modified adeno-associated virus vector and use thereof in treating neurological diseases
AssigneesBingsheng CHANG, Genecombio Ltd.
InventorsAo ZHANG, Zhouteng TAO, Bingsheng CHANG, Guirong DING
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Abstract
Provided in the present application is a nucleic acid fragment which comprises a modified nucleotide sequence encoding an aromatic L-amino acid decarboxylase (AADC). Further provided in the present application are an expression vector comprising the nucleic acid fragment, a recombinant adeno-associated virus (rAAV) comprising the expression vector, a pharmaceutical composition comprising the above-mentioned nucleic acid fragment, expression vector, or rAAV, and the use thereof in treating neurological diseases.
Claims26
A nucleic acid fragment, comprising a nucleotide sequence encoding aromatic L-amino acid decarboxylase (AADC), wherein the nucleotide sequence encoding AADC comprises a nucleotide sequence having at least 80%, 85%, 90%, 95%, 98%, 99%, or 100% identity to the nucleotide sequence shown in SEQ ID NO:
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