发明名称 |
METHODS AND COMPOSITIONS FOR RESTORATION OF CONE FUNCTION IN BCM |
摘要 |
Provided are nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods related to treating cone monochromacies, such as blue cone monochromacy (BCM). Specifically, the nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods involve use of an M-opsin gene operably linked to a cone-specific promoter. Further disclosed is the sequence of a cone-specific PR2.1 promoter. |
申请公布号 |
WO2016149664(A1) |
申请公布日期 |
2016.09.22 |
申请号 |
WO2016US23252 |
申请日期 |
2016.03.18 |
申请人 |
UNIVERSITY OF FLORIDA RESEARCH FOUNDATION, INC. |
发明人 |
PANG, Ji-Jing;HAUSWIRTH, William, W. |
分类号 |
A61K39/12;A61K48/00;C07H21/04;C12N15/00 |
主分类号 |
A61K39/12 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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