| 专利名称 | COMPOSITIONS AND METHODS FOR TREATING CEP290 ASSOCIATED DISEASE | ||
| 申请号 | US16322922 | 申请日 | |
| 公开(公告)号 | US20190169652A1 | 公开(公告)日 | |
| 申请(专利权)人 | EDITAS MEDICINE INC | 发明人 | Morgan Lee MAEDER; Rina J MEPANI; Michael STEFANIDAKIS |
| 专利来源 | 国家知识产权局 | 转化方式 | |
| 摘要 |
Nucleic acids and viral vectors, particularly adeno-associated virus (AAV) vectors are provided that encode Cas9 and paired guide RNAs. The nucleic acids and vectors, and compositions that comprise them, can be used in methods to treat subjects, to alter cells in subjects who may suffer from an inherited retinal dystrophy such as CEP290 associated disease or who may be in need of alteration of a cell or a cellular nucleic acid sequence associated with an inherited retinal dystrophy such as the CEP290 gene, and/or to treat inherited retinal dystrophies including CEP290 associated disease. |
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