公开(公告)号：US20140178942A1

公开(公告)日：2014-06-26

申请号：US14064775

申请日：2013-10-28

Applicant: CELLECTIS

Inventor： ANDRE CHOULIKA ,  ROMAN GALETTO

IPC: C12P19/34

CPC classification number: C12P19/34 ,  A61K38/00 ,  C12N9/22 ,  C12N2740/16022

Abstract: Meganuclease variants which cleave at least one target in the provirus of a retrovirus and in particular which cleave the genomic insertion of the provirus. The present invention particular relates to meganuclease variants which cleave the provirus of the Human Immunodeficiency Virus genome following genomic insertion. Vector encoding such variants, as well as to a cell or multi-cellular organism modified by such a vector and use of said meganuclease variants and derived products for genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy.

Abstract translation: 在逆转录病毒的前病毒中切割至少一个靶，特别是切割前病毒的基因组插入的大范围核酸酶变体。 本发明特别涉及在基因组插入后切割人免疫缺陷病毒基因组的原病毒的大范围核酸酶变体。 编码这样的变体的载体，以及通过这种载体修饰的细胞或多细胞生物体，以及使用所述大范围核酸酶变体和衍生产物用于基因组工程和用于体内和离体（基因细胞治疗）基因组治疗。