MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN INTERLEUKIN-2 RECEPTOR GAMMA CHAIN GENE AND USES THEREOF
    4.
    发明申请
    MEGANUCLEASE VARIANTS CLEAVING A DNA TARGET SEQUENCE FROM THE HUMAN INTERLEUKIN-2 RECEPTOR GAMMA CHAIN GENE AND USES THEREOF 审中-公开
    人类蛋白酶变体清除人类白细胞介素-2受体GAMMA链基因的DNA靶标序列及其用途

    公开(公告)号:US20140017731A1

    公开(公告)日:2014-01-16

    申请号:US13904793

    申请日:2013-05-29

    Applicant: CELLECTIS

    CPC classification number: C12P19/34 C12N9/22

    Abstract: An I-CreI variant, wherein at least one of the two I-CreI monomers has at least two substitutions, one in each of the two functional subdomains of the LAGLIDADG core domain situated respectively from positions 26 to 40 and 44 to 77 of I-CreI, said variant being able to cleave a DNA target sequence from the human IL2RG gene. Use of said variant and derived products for the prevention and the treatment of X-linked severe combined immunodeficiency.

    Abstract translation: I-CreI变体,其中两个I-CreI单体中的至少一个具有至少两个取代,LAGLIDADG核心结构域的两个功能亚结构域中的每一个分别位于I-CreI单体的位置26至40和44至77之间, CreI,所述变体能够从人IL2RG基因切割DNA靶序列。 使用所述变体和衍生产品预防和治疗X连锁严重联合免疫缺陷。

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