公开(公告)号：US09365864B2

公开(公告)日：2016-06-14

申请号：US13900099

申请日：2013-05-22

Applicant: CELLECTIS

Inventor： Jean-Pierre Cabaniols ,  Andre Choulika ,  Christophe Delenda

IPC: C12N15/85 ,  C12N15/90

CPC classification number: C12N15/85 ,  C12N15/902

Abstract: The invention relates to a set of genetic constructs which allow the efficient and reproducible introduction of a specific nucleotide sequence at a fixed position in the genome by generating a double strand break at a specific position in the genome using a meganuclease and so stimulating a homologous recombination event at this locus between the genomic site and a transfected donor sequence. The present invention also relates to methods using these constructs and to these materials in the form of a kit.

公开(公告)号：US20150315557A1

公开(公告)日：2015-11-05

申请号：US14744668

申请日：2015-06-19

Applicant: CELLECTIS

Inventor： Andre Choulika ,  Frederic Cedrone ,  Julianne Smith

IPC: C12N9/22

CPC classification number: C12N9/22 ,  A61K38/00 ,  C12N2710/16622 ,  C12N2730/10122 ,  Y02A50/467

Abstract: An I-CreI variant, wherein at least one of the two I-Cre1 monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the genome of a non-integrating virus, in particular herpes simplex virus (HSV) or Hepatitis B virus (HBV) for use in genome engineering and for in vivo and ex vivo (gene cell therapy) genome therapy as well as the treatment of a virus infection.

Abstract translation: I-CreI变体，其中两个I-Cre1单体中的至少一个具有至少两个取代，位于位于I-CreI的位置26至40和44至77的LAGLIDADG核心结构域的两个功能亚结构域的每一个中的一个 所述变体能够从非整合型病毒，特别是单纯疱疹病毒（HSV）或乙型肝炎病毒（HBV）的基因组中切割DNA靶序列用于基因组工程和体内和离体（基因 细胞治疗）基因组治疗以及病毒感染的治疗。

公开(公告)号：US20140004608A1

公开(公告)日：2014-01-02

申请号：US13900099

申请日：2013-05-22

Applicant: CELLECTIS

Inventor： Jean-Pierre Cabaniols ,  Andre Choulika ,  Christophe Delenda

IPC: C12N15/85

CPC classification number: C12N15/85 ,  C12N15/902

Abstract: The invention relates to a set of genetic constructs which allow the efficient and reproducible introduction of a specific nucleotide sequence at a fixed position in the genome by generating a double strand break at a specific position in the genome using a meganuclease and so stimulating a homologous recombination event at this locus between the genomic site and a transfected donor sequence. The present invention also relates to methods using these constructs and to these materials in the form of a kit.

Abstract translation: 本发明涉及一组基因构建体，其允许通过使用大范围核酸酶在基因组中的特定位置处产生双链断裂并且因此刺激同源重组而在基因组中的固定位置有效且可重复地引入特定核苷酸序列 在该基因座位点和转染供体序列之间的事件。 本发明还涉及以试剂盒形式使用这些构建体和这些材料的方法。