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    METHODS FOR NUCLEAR REPROGRAMMING USING SYNTHETIC TRANSCRIPTION FACTORS
    1.
    发明申请
    METHODS FOR NUCLEAR REPROGRAMMING USING SYNTHETIC TRANSCRIPTION FACTORS 审中-公开
    使用合成转录因子进行核代谢的方法

    公开(公告)号：WO2016201399A1

    公开(公告)日：2016-12-15

    申请号：PCT/US2016/037141

    申请日：2016-06-13

    Applicant: LONZA WALKERSVILLE, INC.

    Inventor： ABRAHAM, Eytan ,  PAYNE, Thomas ,  YOUNG, Robert, J. ,  FRIEDRICH BEN NUN, Inbar

    IPC: C12N5/06 ,  C12N15/85 ,  C12N15/90

    CPC classification number: C12N15/85 ,  A61K35/12 ,  A61K35/15 ,  A61K35/33 ,  A61K35/51 ,  C12N5/0696 ,  C12N2501/40 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2501/608 ,  C12N2506/11 ,  C12N2510/00 ,  G01N33/5073

    Abstract: The current disclosure provides methods for reprogramming mammalian somatic cells by regulating the expression of endogenous cellular genes. Cellular reprogramming of somatic cells can be induced by activating the transcription of embryonic stem cell-associated genes (e.g., oct.3/4 ) and suppressing the transcription of somatic cell-specific and/or cell death-associated genes. The endogenous transcription machinery can be modulated using synthetic transcription factors (activators and suppressors), to allow for faster, and more efficient nuclear reprogramming under conditions amenable for clinical and commercial applications. The current disclosure further provides cells obtained from such methods, along with therapeutic methods for using such cells for the treatment of diseases amendable to stem cell therapy, as well as kits for such uses.

    Abstract translation: 本公开提供了通过调节内源细胞基因的表达来重编程哺乳动物体细胞的方法。 可以通过激活胚胎干细胞相关基因（例如oct.3 / 4）的转录和抑制体细胞特异性和/或细胞死亡相关基因的转录来诱导体细胞的细胞重编程。 可以使用合成转录因子（激活因子和抑制子）调节内源性转录机制，以便在临床和商业应用条件下允许更快，更有效的核重编程。 本公开进一步提供从这些方法获得的细胞以及使用这些细胞治疗可干细胞治疗的疾病的治疗方法以及用于此类用途的试剂盒。

    METHODS AND MEDIA FOR DIFFERENTIATING EYE CELLS
    6.
    发明申请
    METHODS AND MEDIA FOR DIFFERENTIATING EYE CELLS 审中-公开
    用于分化眼细胞的方法和介质

    公开(公告)号：WO2014162040A1

    公开(公告)日：2014-10-09

    申请号：PCT/FI2014/050053

    申请日：2014-01-23

    Applicant: TAMPEREEN YLIOPISTO

    Inventor： MIKHAILOVA, Alexandra ,  ILMARINEN, Tanja ,  SKOTTMAN, Heli

    IPC: C12N5/079 ,  C12N5/0797

    CPC classification number: C12N5/0621 ,  C12N2500/98 ,  C12N2501/11 ,  C12N2501/115 ,  C12N2501/15 ,  C12N2501/40 ,  C12N2501/415 ,  C12N2506/02 ,  C12N2506/03

    Abstract: Here is provided a novel differentiation protocol, which was experimentally shown to give rise to corneal epithelial precursor cells or early pigmented RPE precursor cells in defined and xeno-free conditions. The early precursor cells may be further maturated towards corneal epithelium cells, stratified corneal epithelium or mature RPE cells. Such cells may contribute to treatment and research of corneal and retinal conditions, diseases, pathologies as well as toxicology and drug development.

    Abstract translation: 这里提供了一种新的分化方案，其被实验显示在定义和不含异种病毒条件下产生角膜上皮前体细胞或早期着色的RPE前体细胞。 早期前体细胞可以进一步成熟为角膜上皮细胞，层状角膜上皮或成熟的RPE细胞。 这些细胞可能有助于治疗和研究角膜和视网膜病症，疾病，病理以及毒理学和药物开发。

    A ROLE FOR THE PERLMAN SYNDROME EXONUCLEASE DIS3L2 IN THE LIN28-LET-7 PATHWAY
    7.
    发明申请
    A ROLE FOR THE PERLMAN SYNDROME EXONUCLEASE DIS3L2 IN THE LIN28-LET-7 PATHWAY 审中-公开
    LIN28-LET-7路径中PERLMAN SYNDROME EXONUCLEASE DIS3L2的作用

    公开(公告)号：WO2014144251A1

    公开(公告)日：2014-09-18

    申请号：PCT/US2014/028580

    申请日：2014-03-14

    Applicant: CHILDREN'S MEDICAL CENTER CORPORATION

    Inventor： GREGORY, Richard, I.

    IPC: C12N15/113

    CPC classification number: C12N5/0606 ,  C12N5/0607 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2501/40

    Abstract: The 3 '-5' exonuclease, Dis312, is responsible for the decay of uridylated pre-let-7 miRNA. Biochemical reconstitution assays revealed that 3' oligouridylation stimulates Dis312 activity in vitro, and knockdown of Dis312 in mouse embryonic stem cells leads to the stabilization of pre-let-7 miRNA. These Dis312-depleted stem cells displayed elevated expression of pluripotency genes and delayed differentiation. The present disclosure establishes 3' oligouridylation as an RNA decay signal for Dis312 and identifies the first physiological RNA substrate of this exonuclease.

    Abstract translation: 3'-5'核酸外切酶Dis312负责尿苷酸化的let-7 miRNA的衰变。 生物化学重建测定显示3'寡聚化在体外刺激Dis312活性，并且小鼠胚胎干细胞中Dis312的敲低导致了let-7 miRNA的稳定。 这些Dis312耗尽的干细胞显示多能性基因的表达升高和延迟分化。 本公开建立了3'寡聚化作为Dis312的RNA衰减信号，并鉴定了该外切核酸酶的第一个生理RNA底物。

    COMPOUNDS AND METHODS FOR SELECTIVELY TARGETING CANCER STEM CELLS
    8.
    发明申请
    COMPOUNDS AND METHODS FOR SELECTIVELY TARGETING CANCER STEM CELLS 审中-公开
    用于选择性靶向癌细胞的化合物和方法

    公开(公告)号：WO2014036654A1

    公开(公告)日：2014-03-13

    申请号：PCT/CA2013/050688

    申请日：2013-09-06

    Applicant: MCMASTER UNIVERSITY

    Inventor： BHATIA, Mickie ,  COLLINS, Tony

    IPC: A61K38/12 ,  A61K31/519 ,  A61K31/7048 ,  A61P35/00

    CPC classification number: A61K38/12 ,  A61K31/519 ,  A61K31/7048 ,  C12N5/0693 ,  C12N5/0694 ,  C12N2501/40 ,  C12N2506/30

    Abstract: Described are compounds and methods useful for selectively targeting cancer stem cells. The compounds preferentially induce differentiation and/or reduce the proliferation of cancer stem cells relative to normal stem cells. Compounds useful for selectively targeting cancer stem cells include polyene macrolides such as Nystatin or Amphotericin B, analogs thereof and pharmaceutically acceptable salts thereof.

    Abstract translation: 描述了可用于选择性靶向癌症干细胞的化合物和方法。 化合物相对于正常的干细胞优先诱导分化和/或减少癌症干细胞的增殖。 可用于选择性靶向癌症干细胞的化合物包括多烯大环内酯如制霉菌素或两性霉素B，其类似物和其药学上可接受的盐。

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