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US 2022/0064673 A1Published application
Codon optimized rpgrorf15 genes and uses thereof
Assignee4D Molecular Therapeutics Inc.
InventorsDavid H. KIRN, Melissa A. KOTTERMAN, David SCHAFFER, Peter FRANCIS
Life of this patent
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Abstract
The present disclosure provides codon optimized RPGRorf15 sequences, vectors, and host cells comprising codon optimized RPGRorf15 sequences, and methods of treating retinal disorders such as XLRP comprising administering to the subject a codon optimized RPGRorf15 sequence.
Claims25
A nucleic acid encoding human retinitis pigmentosa GTPase regulator (RPGR) protein of SEQ ID NO:2 and 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 RPGR nucleotide sequence of SEQ ID NO:
- 3 in an otherwise identical cell.
Dependencies are inferred from the claim text.