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AssigneeShanghai Vitalgen Biopharma Co., Ltd.
InventorsYezheng Tao, Shin-Shay Tian, Xiaoping Zhao
Estimating the term…
Provided is a recombinant adeno-associated viral (rAAV) vector comprising one or two of (a) to (c): (a) a nucleotide sequence encoding aromatic L-amino acid decarboxylase (AADC), (b) a nucleotide sequence encoding glucocerebrosidase (GBA1); and (c) a nucleotide sequence encoding a neurotrophic factor (NTF), such as cerebral dopamine neurotrophic factor (CDNF) or glial cell derived neurotrophic factor (GDNF), for treating neurodegenerative disorders, particularly Parkinson's disease (PD), Multiple system atrophy (MSA), Gaucher's disease (GD), and other proteinopathies. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the viral particles, and uses thereof.
An isolated nucleic acid molecule, comprising a first nucleotide sequence encoding a first protein operatively linked to a second nucleotide sequence encoding a second protein, wherein the first protein and the second protein are selected from the following combination (a) or (b):
A codon-optimized sequence of AADC, having a nucleotide as shown in any one of SEQ ID NOs:
Dependencies are inferred from the claim text.