Sequential gene editing in primary immune cells

    公开(公告)号:US11466291B2

    公开(公告)日:2022-10-11

    申请号:US16314697

    申请日:2017-06-30

    Applicant: CELLECTIS

    Abstract: The invention pertains to the field of adaptive cell immunotherapy. It aims at reducing the occurrence of translocations and cell deaths when several specific endonuclease reagents are used altogether to genetically modify primary immune cells at different genetic loci. The method of the invention allows to yield safer immune primary cells harboring several genetic modifications, such as triple or quadruple gene inactivated cells, from populations or sub-populations of cells originating from a single donor or patient, for their subsequent use in therapeutic treatments.

    METHOD FOR THE GENERATION OF COMPACT TALE-NUCLEASES AND USES THEREOF
    8.
    发明申请
    METHOD FOR THE GENERATION OF COMPACT TALE-NUCLEASES AND USES THEREOF 审中-公开
    用于生成紧凑型陶瓷核的方法及其用途

    公开(公告)号:US20160298098A1

    公开(公告)日:2016-10-13

    申请号:US15069672

    申请日:2016-03-14

    Abstract: The present invention relates to a method for the generation of compact Transcription Activator-Like Effector Nucleases (TALENS) that can efficiently target and process double-stranded DNA. More specifically, the present invention concerns a method for the creation of TALENs that consist of a single TALE DNA binding domain fused to at least one catalytic domain such that the active entity is composed of a singe polypeptide chain for simple and efficient vectorization and does not require dimerization to target a specific single double-stranded DNA tar et sequence of interest and process DNA nearby said DNA target sequence. The present invention also relates to compact TALENs, vectors, compositions and kits used to implement the method.

    Abstract translation: 本发明涉及能够有效靶向和加工双链DNA的致密转录激活子样效应核酸酶(TALENS)的生成方法。 更具体地,本发明涉及一种用于产生TALEN的方法,其由与至少一个催化结构域融合的单个TALE DNA结合结构域组成,使得活性实体由简单且有效的载体化的单个多肽链组成,并且不 需要二聚化来靶向目标特定的单链双链DNA焦油和序列,并在DNA附近处理DNA靶序列。 本发明还涉及用于实施该方法的紧凑型TALEN,载体,组合物和试剂盒。

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