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26 条记录 (耗时 0.027 秒)

    CAS9 NUCLEASE PLATFORM FOR MICROALGAE GENOME ENGINEERING
    11.
    发明申请
    CAS9 NUCLEASE PLATFORM FOR MICROALGAE GENOME ENGINEERING 审中-公开
    用于微藻基因工程的CAS9核平台

    公开(公告)号：US20160304893A1

    公开(公告)日：2016-10-20

    申请号：US15103773

    申请日：2014-12-12

    申请人： CELLECTIS

    发明人： Fayza DABOUSSI ,  Marine BEURDELEY ,  Alexandre JUILLERAT

    IPC分类号： C12N15/82 ,  C12N9/22

    CPC分类号： C12N15/8213 ,  C12N9/22

    摘要： The present invention relates to a method of genome engineering in microalgae using the Cas9/CRISPR system. In particular, the present invention relates to methods of delivering RNA guides via cell penetrating peptides in microalgae, preferably in stable integrated Cas9 microalgae. The present invention also relates to kits and isolated cells comprising Cas9, split Cas9 or guide RNA and Cas9-fused cell-penetrating peptides. The present invention also relates to isolated cells obtained by the methods of the invention.

    摘要翻译： 本发明涉及使用Cas9 / CRISPR系统的微藻中的基因组工程方法。 特别地，本发明涉及通过微藻中的细胞穿透肽递送RNA引导物的方法，优选在稳定的综合Cas9微藻中。 本发明还涉及包含Cas9，裂解Cas9或引导RNA和Cas9融合的细胞穿透肽的试剂盒和分离的细胞。 本发明还涉及通过本发明的方法获得的分离的细胞。

    DESIGN OF RARE-CUTTING ENDONUCLEASES FOR EFFICIENT AND SPECIFIC TARGETING DNA SEQUENCES COMPRISING HIGHLY REPETITIVE MOTIVES
    12.
    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。

    TAL- effector nuclease (TALEN) -MODIFIED ALLOGENIC CELLS SUITABLE FOR THERAPY
    15.
    发明申请
    TAL- effector nuclease (TALEN) -MODIFIED ALLOGENIC CELLS SUITABLE FOR THERAPY 有权

    公开(公告)号：US20230138915A1

    公开(公告)日：2023-05-04

    申请号：US16340412

    申请日：2017-10-19

    申请人： CELLECTIS

    发明人： Philippe DUCHATEAU ,  Brian BUSSER ,  Alexandre JUILLERAT ,  Anne-Sophie GAUTRON ,  Laurent POIROT

    IPC分类号： C12N15/90 ,  C12N15/11 ,  C12N9/22 ,  C12N5/0783 ,  C07K14/725

    摘要： The invention relates to the fields of immunotherapy, molecular biology and recombinant nucleic acid technology. In particular, the invention relates to a TALEN-modified human primary cell comprising in its genome, a modified human T cell receptor alpha gene with an insertion comprising at least, from 5′ to 3′, a polynucleotide encoding a self-cleaving peptide, a chimeric antigen receptor, wherein the cell has undetectable cell-surface expression of the endogenous alpha beta T cell receptor as compared to a TCR positive control cell and expresses a receptor to target a pathological cell, use of said cell for treating a disease, including cancer. The invention further relates to methods for producing such a TALEN-modified cell, and to means for detecting such an engineered human primary cell or other genetically modified human primary cell obtained using alternative and/or additional rare cutting endonucleases.