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US 11,357,870 B2Granted
Codon optimized REP1 genes and uses thereof
Assignee4D Molecular Therapeutics Inc.
InventorsDavid H. Kirn, Melissa A. Kotterman, Peter Francis
Life of this patent
Estimating the term…
Abstract
The present disclosure provides codon optimized nucleotide sequences encoding human REP1, vectors, and host cells comprising codon optimized REP1 sequences, and methods of treating retinal disorders such as choroideremia comprising administering to the subject a codon optimized sequence encoding human REP1.
Claims25
A nucleic acid encoding human Rab escort protein-1 (REP1) 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 REP1 nucleotide sequence of SEQ ID NO:
- 3 in an otherwise identical cell.
Dependencies are inferred from the claim text.