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US 2025/0064982 A1Published application
Recombinant aav vectors for treating glutaric aciduria type i
AssigneeShanghai Vitalgen Biopharma Co., Ltd.
InventorsLu GUO, Xi ZHU, Shin-Shay TIAN, Xiaoping ZHAO
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Abstract
The present disclosure relates to codon-optimized sequences coding for hGCDH polypeptide and recombinant adeno-associated virus (rAAV) vectors comprising one of said sequences under the control of a promoter component. Also provided herein are viral particles comprising the rAAV vector, a pharmaceutical composition comprising the rAAV vector or the viral particles, and uses thereof in treating Glutaric aciduria type I (GA-I).
Claims20
An isolated nucleic acid molecule, comprising a nucleotide sequence selected from a group of sequences consisting of SEQ ID NOs:
- 11-18, wherein the nucleotide sequence encodes human glutaryl-CoA dehydrogenase (hGCDH) polypeptide having an amino acid sequence as shown in SEQ ID NO:
- 33.
Dependencies are inferred from the claim text.