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CODON OPTIMIZED NUCLEIC ACID ENCODING A RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR)
专利权人:
Applied Genetic Technologies Corporation
发明人:
YE, Guo-Jie,LIU, Jilin
申请号:
EP20150779735
公开号:
EP3132051(A1)
申请日:
2015.04.15
申请国别(地区):
欧洲专利局
年份:
2017
代理人:
摘要:
This invention relates generally to a codon optimized nucleic acid encoding a retinitis pigmentosa GTPase regulator (RPGR) protein. The nucleic acid has enhanced stability during plasmid production relative to a wildtype cDNA encoding the RPGR protein. The invention also relates to expression cassettes, vectors, and host cells comprising the codon optimized nucleic acid. Methods for preparing a recombinant adeno-associated (rAAV) expression vector comprising the codon optimized nucleic acid sequence are also provided. The nucleic acids, expression cassettes, vectors, and host cells provided may be useful in the large scale production of rAAV expression vectors for gene therapy applications.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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