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Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
专利权人:
Applied Genetic Technologies Corporation
发明人:
Ye, Guo-Jie
申请号:
AU2014265799
公开号:
AU2014265799A1
申请日:
2014.05.05
申请国别(地区):
AU
年份:
2015
代理人:
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.
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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