ENDOGENOUS RETROVIRUS TRANSCRIPTION AS A MARKER FOR PRIMATE NAÏVE PLURIPOTENT STEM CELLS
    41.
    发明申请
    ENDOGENOUS RETROVIRUS TRANSCRIPTION AS A MARKER FOR PRIMATE NAÏVE PLURIPOTENT STEM CELLS 审中-公开
    作为初始标记的内源性逆转录酶转录物可产生大量干细胞

    公开(公告)号:WO2016055519A1

    公开(公告)日:2016-04-14

    申请号:PCT/EP2015/073144

    申请日:2015-10-07

    Abstract: The present invention relates to the use of one or more type 7 long terminal repeat (LTR7) nucleic acid sequences of type H human endogenous retroviruses (HERVH) ("LTR7/HERVH nucleic acid sequences") for identifying primate naive pluripotent stem cells. The invention is directed to the use of LTR7/HERVH nucleic acid sequences as a marker, wherein LTR7/HERVH-associated transcription is used as a marker for primate naive pluripotent stem cells. The invention also relates to a reporter construct comprising LTR7/HERVH nucleic acid sequences in addition to the use of said reporter, in particular for optimizing culture conditions for naïve primate pluripotent stem cells. The invention also relates to a cell growth medium for cultivation of primate naive pluripotent stem cells that preferably exhibit elevated levels of LTR7/HERVH-associated transcription in comparison to control cells.

    Abstract translation: 本发明涉及H型人内源性逆转录病毒(HERVH)(“LTR7 / HERVH核酸序列”)的一种或多种7型长末端重复序列(LTR7)核酸序列用于鉴定灵长类天真多能干细胞的用途。 本发明涉及使用LTR7 / HERVH核酸序列作为标记物,其中LTR7 / HERVH相关转录用作灵长类动物幼稚多能干细胞的标记物。 除了使用所述报道基因之外,本发明还涉及包含LTR7 / HERVH核酸序列的报告基因构建体,特别是用于优化初始灵长类动物多能干细胞的培养条件。 本发明还涉及用于培养灵长类动物幼体多能干细胞的细胞生长培养基,与对照细胞相比,其优选表现出升高的LTR7 / HERVH相关转录水平。

    METHODS AND COMPOSITIONS FOR MODULATING NUCLEASE-MEDIATED GENOME ENGINEERING IN HEMATOPOIETIC STEM CELLS
    42.
    发明申请
    METHODS AND COMPOSITIONS FOR MODULATING NUCLEASE-MEDIATED GENOME ENGINEERING IN HEMATOPOIETIC STEM CELLS 审中-公开
    在造血干细胞中调节核酸介导的基因工程的方法和组合物

    公开(公告)号:WO2016014794A1

    公开(公告)日:2016-01-28

    申请号:PCT/US2015/041729

    申请日:2015-07-23

    Abstract: The present disclosure is in the field of genome engineering, particularly targeted modification of the genome of a hematopoietic stem cell. The present invention describes compositions and methods for use in gene therapy and genome engineering, particularly of hematopoietic cells, including HSCs. The present inventors have determined that certain culture conditions can impact the efficiency and the nature of gene modification after a double strand break (independent of the nuclease used), particularly in LT-HSCs.

    Abstract translation: 本公开在基因组工程领域,特别是造血干细胞基因组的靶向修饰。 本发明描述了用于基因治疗和基因组工程,特别是造血细胞(包括HSC)的组合物和方法。 本发明人已经确定某些培养条件可以影响双链断裂后的基因修饰的效率和性质(与使用的核酸酶无关),特别是在LT-HSC中。

    METHODS AND COMPOSITIONS FOR ENCHANCING TRANSDUCTION EFFICIENCY OF RETROVIRAL VECTORS
    46.
    发明申请
    METHODS AND COMPOSITIONS FOR ENCHANCING TRANSDUCTION EFFICIENCY OF RETROVIRAL VECTORS 审中-公开
    提高转染载体传递效率的方法和组合

    公开(公告)号:WO2014109728A1

    公开(公告)日:2014-07-17

    申请号:PCT/US2013/000136

    申请日:2013-05-17

    Abstract: The present invention provides methods for enhancing transduction efficiency of a viral vector into a host cell such as a stem cell. The methods involve transducing the host cell with the vector in the presence of an inhibitor of mTOR complexes (e.g., rapamycin or analog compound thereof). Also provided in the invention are kits or pharmaceutical combinations for delivering a therapeutic agent into a target cell with enhanced targeting frequency and payload delivery. The kits or pharmaceutical combinations typically contain a viral vector encoding the therapeutic agent, and an inhibitor of mTOR complexes.

    Abstract translation: 本发明提供了用于增强病毒载体转导入宿主细胞如干细胞的转导效率的方法。 所述方法包括在mTOR复合物抑制剂(例如雷帕霉素或其类似物)存在下用载体转导宿主细胞。 本发明还提供了用于将治疗剂递送到具有增强的靶向频率和有效载荷递送的靶细胞中的试剂盒或药物组合。 试剂盒或药物组合通常含有编码治疗剂的病毒载体和mTOR复合物的抑制剂。

    IMPROVED CULTURE MEDIA FOR EMBRYONIC CELLS
    48.
    发明申请
    IMPROVED CULTURE MEDIA FOR EMBRYONIC CELLS 审中-公开
    改进的胚胎培养基培养基

    公开(公告)号:WO2013149957A1

    公开(公告)日:2013-10-10

    申请号:PCT/EP2013/056779

    申请日:2013-03-28

    Inventor: WÅNGGREN, Kjell

    CPC classification number: C12N5/0604 C12N15/873 C12N2501/2306 C12N2501/235

    Abstract: A composition and method for in vitro fertilization is provided which uses culture media comprising sgp 130. Embryo culture media that include sgp 130 are able to provide an improved blastocyst development rate as well increasing the proportion of high quality blastocysts.

    Abstract translation: 提供了一种用于体外受精的组合物和方法,其使用包含sgp 130的培养基。包含sgp 130的胚胎培养基能够提供改善的胚泡发育速率,并且增加高质量囊胚的比例。

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