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US 2024/0309068 A1Published application
Recombinant adeno-associated viral vectors for treating bietti crystalline dystrophy
AssigneeShanghai Vitalgen Biopharma Co., Ltd.
InventorsLu GUO, Yezheng TAO, Shin-Shay TIAN, Bin QU, Wei LI, Xi ZHU, Xiaoping ZHAO
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Abstract
The present disclosure provides a recombinant adeno-associated vector comprising a codon-optimized sequence encoding CYP4V2 linked to selected gene expression regulatory sequences and its use in treating Bietti Crystalline Dystrophy (BCD).
Claims16
An isolated nucleic acid molecule, comprising a nucleotide sequence which is at least one of SEQ ID NOs:
- 2-17 and that encodes human CYP4V2 polypeptide.
Dependencies are inferred from the claim text.