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US 12,371,474 B2Granted
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
Life of this patent
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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).
Claims15
An isolated nucleic acid molecule, comprising the nucleotide sequence of SEQ ID NO:
- 8, 9, 15, 16 or 17, and that encodes human CYP4V2 polypeptide.
Dependencies are inferred from the claim text.