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US 2021/0332341 A1Published application
Codon optimized gla genes and uses thereof
Assignee4D Molecular Therapeutics Inc.
InventorsDavid H. KIRN, Melissa KOTTERMAN, Peter FRANCIS, David SCHAFFER, Paul SZYMANSKI, Kevin WHITTLESEY
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Abstract
The present disclosure provides codon optimized nucleotide sequences encoding human alpha-galactosidase A, vectors, and host cells comprising codon optimized alpha-galactosidase A sequences, and methods of treating disorders such as Fabry disease comprising administering to the subject a codon optimized sequence encoding human alpha-galactosidase A.
Claims25
A nucleic acid encoding human α-galactosidase A (AGA) protein codon optimized for expression in humans, the nucleic acid comprising the nucleotide sequence set forth as SEQ ID NO:
- 1 or comprising a nucleotide sequence at least 95% identical thereto, wherein the nucleic acid is expressed at a greater level compared with the level of expression of the wild type GLA nucleotide sequence of SEQ ID NO:
- 3 in an otherwise identical cell.
Dependencies are inferred from the claim text.