基本信息:
- 专利标题： COMPOSITIONS AND PRODUCTION OF NICKED CLOSED-ENDED DNA VECTORS
- 申请号：PCT/US2020/042445 申请日：2020-07-17
- 公开(公告)号：WO2021011840A1 公开(公告)日：2021-01-21
- 发明人： ALKAN, Ozan , KOTIN, Robert, Michael , KERR, Douglas, Anthony , MONDS, Russell , PELLETIER, Carolyn , STANTON, Matthew
- 申请人： GENERATION BIO CO.
- 申请人地址： 301 Binney Street, 4th Floor Cambridge, MA 02142 US
- 专利权人： GENERATION BIO CO.
- 当前专利权人： GENERATION BIO CO.
- 当前专利权人地址： 301 Binney Street, 4th Floor Cambridge, MA 02142 US
- 代理机构： CLARKE, Marcie, B. et al.
- 优先权： US62/875,262 20190717
- 主分类号： C12N15/63
- IPC分类号： C12N15/63 ; C12N15/09 ; C12N15/85
摘要：
The present application discloses methods for synthetic production and cell-free synthesis of DNA vectors, particularly closed-ended linear DNA vectors having one or more gaps ( e.g ., nicked ceDNA vectors, "neDNA") and adenoassociated-virus (AAV) vector which is single strand DNA having linear and continuous structure, for delivery and expression of a transgene in the host cell. The present invention also relates to an in vitro process for production of closed-ended DNA vectors, corresponding DNA vector products produced by the methods and uses thereof, and oligonucleotides and kits useful in the process of the present invention.