治療視網膜營養性萎縮之病毒載體农业专利-农业专业知识服务系统

发明人:: BIGELOW, CHAD ERIC,比吉罗　查德　艾瑞克,比吉羅　查德　艾瑞克,CHOI, VIVIAN,卓依　维维安,卓依　維維安,DRYJA, THADDEUS PETER,卓贾　撒德斯　彼得,卓賈　撒德斯　彼得,POLICE, SESHIDHAR REDDY,波利仕　撒希德尔　罗迪,波利仕　撒希德爾　羅迪,比吉罗查德艾瑞克,比吉羅查德艾瑞克,卓依维维安,卓依維維安,卓贾撒德斯彼得,卓賈撒德斯彼得,波利仕撒希德尔罗迪,波利仕撒希德爾羅迪

摘要：: The present invention relates to viral vectors that are capable of delivering a heterologous gene to the retina and in particular delivering RLBP1 to RPE and Müller cells of the retina. The invention also relates nucleic acids useful for producing viral vectors, compositions comprising the viral vectors and uses of the compositions and viral vectors. The invention also relates to methods of delivering and/or expressing a heterologous gene to the retina, improving the rate of dark adaption in a subject and treating RLBP1-associated retinal dystrophy.本發明係關於能夠傳遞異源基因至視網膜且詳言之傳遞RLBP1至視網膜之RPE及穆勒細胞(Müller cell)之病毒載體。本發明亦關於適用於產生病毒載體之核酸、包含該等病毒載體之組合物及該等組合物及病毒載體之用途。本發明亦關於傳遞及/或表現異源基因至視網膜、提高個體之暗適應速率及治療RLBP1相關視網膜營養性萎縮之方法。