University of Florida Research Foundation; Incorporated
发明人:
William W. Hauswirth,Ji-Jing Pang
申请号:
US15559315
公开号:
US20180112231A1
申请日:
2016.03.18
申请国别(地区):
US
年份:
2018
代理人:
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.