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    GENE THERAPY FOR THE TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) RELATED TO RAG1
    1.
    发明公开
    GENE THERAPY FOR THE TREATMENT OF SEVERE COMBINED IMMUNODEFICIENCY (SCID) RELATED TO RAG1 审中-公开

    公开(公告)号：US20240318154A1

    公开(公告)日：2024-09-26

    申请号：US18561938

    申请日：2022-05-20

    申请人： CELLECTIS ,  Albert-Ludwigs-Universitat Freiburg

    发明人： Toni CATHOMEN ,  Tatjana CORNU ,  Julia KLERMUND ,  Manuel RHIEL ,  Julia ROSITZKA ,  Philippe DUCHATEAU ,  Alexandre JUILLERAT

    IPC分类号： C12N9/22 ,  A61K35/28 ,  C07K14/47 ,  C12N5/0789 ,  C12N15/90

    CPC分类号： C12N9/22 ,  A61K35/28 ,  C07K14/4705 ,  C12N5/0647 ,  C12N15/907 ,  C12N2510/00

    摘要： The present invention generally relates to the field of genome engineering (gene editing), and more specifically to gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1. Particularly, the present invention pertains to the treatment of RAG1 deficiency in long-term repopulating hematopoietic stem cells (HSCs). The present invention provides means and methods for genetically modifying HSCs involving gene editing reagents, such as TALE-nucleases, that specifically target a non-functional endogenous RAG1 gene, comprising at least one mutation causing Severe Combined Immunodeficiency (SCID), thereby allowing the restoration of the normal cellular phenotype. The present invention also provides engineered RAG1-edited HSCs comprising an exogenous sequence comprising a nucleic acid sequence encoding a functional RAG1 protein which is integrated in said HSCs' genome into a non-functional RAG1 endogenous locus, resulting in the expression of a functional RAG1 polypeptide. The present invention further provides populations of cells comprising said engineered HSCs, pharmaceutical compositions comprising said engineered HSCs or populations of cells, as well as their use in gene therapy for the treatment of Severe Combined Immunodeficiency (SCID) related to RAG1.

    MICELLE BASED SYSTEM NUCLEASE ENCAPSULATION FOR IN-VIVO GENE EDITING
    4.
    发明申请
    MICELLE BASED SYSTEM NUCLEASE ENCAPSULATION FOR IN-VIVO GENE EDITING 审中-公开

    公开(公告)号：US20190032088A1

    公开(公告)日：2019-01-31

    申请号：US16073778

    申请日：2017-02-24

    申请人： CELLECTIS

    发明人： Philippe DUCHATEAU ,  Veronique ZENNOU

    IPC分类号： C12N15/88 ,  A61K9/00 ,  C12N9/22 ,  C12N15/11 ,  A61K48/00 ,  A61K9/127

    摘要： The invention pertains to therapies that require gene editing, and more specifically to non-viral methods for in vivo delivery of endonuclease reagents to specific tissues or cells. According to the invention, the endonuclease reagents are encapsulated into micelle structures of 50 to 150 nm diameter for intravenous injection. The invention thus provides therapeutic composition including such micelles structures, by which endonuclease reagents can be released into cell under RNA form for their use in the treatment of gene related diseases.

    NEW METHOD OF SELECTION OF ALGAL-TRANSFORMED CELLS USING NUCLEASE
    8.
    发明申请
    NEW METHOD OF SELECTION OF ALGAL-TRANSFORMED CELLS USING NUCLEASE 审中-公开
    使用核酸选择ALGAL转化细胞的新方法

    公开(公告)号：US20160312233A1

    公开(公告)日：2016-10-27

    申请号：US15103751

    申请日：2014-12-12

    申请人： CELLECTIS

    发明人： Philippe DUCHATEAU ,  Fayza DABOUSSI

    IPC分类号： C12N15/79 ,  C12N15/90 ,  C12N9/22 ,  C12N15/10

    CPC分类号： C12N15/79 ,  C12N9/22 ,  C12N15/102 ,  C12N15/8209 ,  C12N15/821 ,  C12N15/8213 ,  C12N15/902 ,  C12N2800/80 ,  C12Y301/00

    摘要： The invention relates to a method to select transformed cells. In particular, the present invention relates to the use of a nuclease engineered to inactivate selectable marker which confers cell resistance to a toxic compound. The present invention relates to methods of modifying genome of a cell, preferably an algal cell comprising the present selection step. The present invention also relates to specific engineered nucleases, polynucleotides, vectors encoding thereof, kits and isolated cells comprising said nuclease.

    摘要翻译： 本发明涉及一种选择转化细胞的方法。 特别地，本发明涉及工程化的核酸酶在使有毒化合物赋予细胞耐药性的选择性标记失活的用途。 本发明涉及修饰细胞基因组的方法，所述方法优选包含本选择步骤的藻细胞。 本发明还涉及特异性工程化核酸酶，多核苷酸，编码它们的载体，试剂盒和包含所述核酸酶的分离的细胞。

    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES
    9.
    发明申请
    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES 审中-公开
    用于有效和特异性靶向包含高度重复活性的DNA序列的切割内切酶的设计

    公开(公告)号：US20160273002A1

    公开(公告)日：2016-09-22

    申请号：US15031996

    申请日：2014-10-24

    申请人： CELLECTIS

    发明人： Philippe DUCHATEAU ,  Alexandre JUILLERAT

    IPC分类号： C12N15/90 ,  C07K14/195 ,  C12N9/22

    CPC分类号： C12N15/907 ,  A61K38/00 ,  A61K48/00 ,  A61K48/005 ,  C07K14/195 ,  C07K2319/80 ,  C12N9/22 ,  C12Y301/00 ,  C12Y301/21004

    摘要： The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.

    摘要翻译： 本发明在遗传编辑工具和基因工程方法领域。 它涉及设计用于在染色体中收缩高度重复动机的稀有内切核酸酶的工程，这些动机是某些遗传疾病的起源，特别是所谓的“三重重复疾病”，如亨廷顿疾病。 本发明包括用于收缩重复动机的方法，用于在重复病症的基因中重复动作的稀有内切核酸酶，编码它们的多核苷酸和载体以及所得药物组合物。