Codon-optimized nucleic acid encoding retinitis pigmentosa GTPase regulator (RPGR)

摘要

The present invention relates generally to codon optimized nucleic acids encoding retinitis pigmentosa GTPase regulator (RPGR) proteins. This nucleic acid has increased stability during plasmid production compared to the wild type cDNA encoding the RPGR protein. The invention also relates to expression cassettes, vectors, and host cells comprising codon optimized nucleic acids. A method of preparing a recombinant adeno-associated (rAAV) expression vector comprising a codon optimized nucleic acid sequence is also provided. The provided nucleic acids, expression cassettes, vectors, and host cells can be useful for large-scale production of rAAV expression vectors for gene therapy applications. [Selection figure] None