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1. 发明申请

WO2012094560A2 PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES 审中-公开
标题翻译：促进剂，表达CASSETTES，载体，作用和治疗感染和其他疾病的方法
公开(公告)号：WO2012094560A2
公开(公告)日：2012-07-12
申请号：PCT/US2012/020423
申请日：2012-01-06
申请人： APPLIED GENETIC TECHNOLOGIES CORPORATION , YE, Guo-Jie , CHULAY, Jeffrey, D.
发明人： YE, Guo-Jie , CHULAY, Jeffrey, D.
IPC分类号： C12N15/11 , C12N15/861 , C12N15/12 , A61K48/00
CPC分类号： C12N15/85 , A61K48/0058 , C07K14/705 , C12N2799/025 , C12N2830/008
摘要： The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of genetic diseases that affect the cone cells of the retina.
摘要翻译：本发明提供分离的启动子，转基因表达盒，载体，试剂盒和用于治疗影响视网膜的锥细胞的遗传疾病的方法。

2. 发明申请

WO2014186160A1 PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE THREATMENT OF ACHROMATOPSIA AND OTHER DISEASES 审中-公开
标题翻译：促销剂，表达CASSETTES，矢车菊，搭便车及其他疾病的治疗方法
公开(公告)号：WO2014186160A1
公开(公告)日：2014-11-20
申请号：PCT/US2014/036792
申请日：2014-05-05
申请人： APPLIED GENETIC TECHNOLOGIES CORPORATION
发明人： YE, Guo-Jie
IPC分类号： A61K48/00
CPC分类号： A61K48/00 , A61K48/0058 , C12N15/86 , C12N2750/14141 , C12N2830/008
摘要： The present invention provides isolated promoters, transgene expression cassettes, vectors, kits, and methods for treatment of genetic diseases that affect the cone cells of the retina. The present invention features, in a first aspect, a nucleic acid comprising a portion of the cone cell specific promoter PR 2.1. In one embodiment, the nucleic acid comprises the sequence SEQ ID NO: 4. In another embodiment, PR2.1 is truncated at the 5' or the 3' end. In one embodiment of the above aspects, the promoter is capable of promoting CNGB3 expression in S-cone cells, M-cone cells, and L-cone cells. In another embodiment of the above aspects, the promoter is capable of promoting CNGA3 expression in S-cone cells, M-cone cells, and L-cone cells. In yet another embodiment of the above aspects, the promoter is capable of promoting GNAT2 expression in S-cone cells, M-cone cells, and L-cone cells.
摘要翻译：本发明提供分离的启动子，转基因表达盒，载体，试剂盒和用于治疗影响视网膜的锥细胞的遗传疾病的方法。在第一方面，本发明的特征在于包含一部分锥细胞特异性启动子PR 2.1的核酸。在一个实施方案中，核酸包含序列SEQ ID NO：4。在另一个实施方案中，PR2.1在5'或3'末端截短。在上述方面的一个实施方案中，启动子能够促进S-锥细胞，M锥细胞和L-锥细胞中的CNGB3表达。在上述方面的另一个实施方案中，启动子能够促进S-锥细胞，M-锥细胞和L-锥细胞中的CNGA3表达。在上述方面的另一个实施方案中，启动子能够促进S锥体细胞，M锥细胞和L-锥细胞中的GNAT2表达。

3. 发明申请

WO2015160893A1 CODON OPTIMIZED NUCLEIC ACID ENCODING A RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR) 审中-公开
标题翻译： CODON优化核酸编码重组腺嘌呤激酶调节因子（RPGR）
公开(公告)号：WO2015160893A1
公开(公告)日：2015-10-22
申请号：PCT/US2015/025887
申请日：2015-04-15
申请人： APPLIED GENETIC TECHNOLOGIES CORPORATION
发明人： YE, Guo-Jie
IPC分类号： C12P21/06 , C12N15/64 , C12N15/00 , C12N9/00
CPC分类号： C07K14/4702 , C12N7/00 , C12N9/00 , C12N15/00 , C12N15/64 , C12N15/68 , C12N15/86 , C12N2710/16621 , C12N2750/14143 , C12N2830/008
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.
摘要翻译：本发明一般涉及编码色素性视网膜炎GTP酶调节蛋白（RPGR）蛋白质的密码子优化的核酸。相对于编码RPGR蛋白的野生型cDNA，核酸在质粒生产过程中具有增强的稳定性。本发明还涉及包含密码子优化的核酸的表达盒，载体和宿主细胞。还提供了制备包含密码子优化的核酸序列的重组腺相关（rAAV）表达载体的方法。提供的核酸，表达盒，载体和宿主细胞可用于大规模生产用于基因治疗应用的rAAV表达载体。

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